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Diabetic foot wounds kill millions, but high-tech solutions and teamwork are making a difference – Thehour.com

(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)

David G. Armstrong, University of Southern California

(THE CONVERSATION) What if someone told you that theres a disease you could catch where you couldnt feel any symptoms coming on? And that this occurs every 1.2 seconds somewhere in the world?

What if you were stricken with this disease then there would be a 5% chance youd lose a limb within a year and a 50-70% chance youd be dead in five years? What if you were told that this problem cost more than the five most expensive cancers in the U.S. but far less than one one-thousandth of comparative federal and private funding is spent on attacking it?

Ladies and gentlemen, please allow me to introduce you to the humble diabetic foot ulcer. While the problem may strike at the end of the body, far away from the heart or the brain, its effects are far-reaching.

I have spent my career treating and researching the lower extremity complications of diabetes. Based on my research and experience, I believe our society could eliminate immeasurable suffering if we collectively paid more attention to this problem.

How do diabetic foot ulcers occur?

OK, I know this isnt a sexy topic. Foot wounds are ugly. Many people who have them are poor. But bear with me. They are a reality for far too many Americans and people across the globe. The ages of these patients are bimodal, in that there is one population of people who are old and getting older. Conversely, with more and more people being diagnosed with Type 2 diabetes earlier, there is a population that is younger than ever being afflicted with wounds, infections and amputation. Ignoring the problem is an example of ignoring the needs of a silent and vulnerable population.

About 31 million people in the U.S. have diabetes, and about half a billion worldwide.

Diabetic foot ulcers develop because people with diabetes slowly lose the gift of pain. Over many years, people with diabetes lose feeling in their extremities. This occurs first and generally most profoundly in their feet.

Once this occurs, people with diabetes might wear a hole in their foot, just as you or I might wear a hole in a sock or shoe. This hole is called a diabetic foot ulcer.

About half the time, the ulcer will become infected. This increases the risk of further tissue damage and, in the face of frequent vascular disease, high-level amputation. Often all of this occurs with few, if any, symptoms until it is too late.

Solutions and hope

There is also good news. Studies have suggested that high-level amputations seem to decrease when interdisciplinary care is in place, regardless of the country.

Interdisciplinary teams consist of podiatric and vascular surgeons, the so-called Toe and Flow model. The concept is simple; these two specialists, can manage a great deal of the medical, surgical and biomechanical aspects of healing and aftercare.

When we add core physical therapy to this, then the threesome (what we in the field call Toe, Flow and Go) is really quite formidable. For example, our clinics at the University of Southern California and Rancho Los Amigos in Los Angeles have active participation from more than eight specialists ranging from plastic surgery to prosthetics/orthotics, to occupational therapy to nutrition to general practice to infectious disease to diabetology to nurse case management.

Truly, it takes a village to preserve a limb.

Smart boots, high-tech vacuums and sheets of stems cells

It has long been said in wound care that its not what one puts on a wound that heals it, but what one takes off. That maxim is absolutely true in the diabetic foot. Protection of the wound is key.

The gold standard for protecting the wound has been, believe it or not, to put the patient into a special cast. This device works so well because it protects the foot in a process known as offloading, or taking the burden off the foot. By its design, this cast is not easy to remove.

While this has been my personal favorite device to heal these foot wounds, patients dont like it and most doctors dont, either. In fact, fewer than 2% of centers in the country use this as their primary means of offloading. Reasons for this include fear of putting an open wound into a cast (even though the data largely refute this), the time required to apply and remove it and patients being miserable in a hot and heavy device.

Very recently, tech company offshoots have begun to partner with prosthetic/orthotic companies to create next-gen devices that can coax patients into wearing their protective device rather than forcing it upon them. They are using phone calls and a smartwatch.

After focusing on offloading pressure, the next question is what can be done to heal the wound.

Technologies ranging from fancy vacuums, to donated placental tissue, to repurposing blood cells into a dressing to topical oxygen systems have shown recent promise. Active research is being conducted with stem cell sheets consisting of specialized cells seeded on a clear sheet, spread-on skin, and gene therapy.

Remission

As challenging as healing the wound heals, the real challenge is whats next. Following healing, 40% of foot wounds will recur in one year, about two-thirds at three years, and nearly three out of four at five years.

At USC, along with colleagues in the National Health Service in the U.K., we have developed remission clinics designed to extend and promote an active life for this high-risk patient population.

This has also been combined with things like smart insoles, socks and home-based bathmats that can identify wounds before they occur. These technologies will likely initially be subscription-based but may expand beyond that.

Diabetic foot ulcers are common, complex and costly. Theyre sinister in that they come on quietly. Perhaps, though, it is now up to us to alert our own families, communities and leaders to this condition. It is, I believe, only by teaming up that we can stem the tide and preserve not only limbs, but extend lifespan, healthspan and hope.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

This article is republished from The Conversation under a Creative Commons license. Read the original article here: http://theconversation.com/diabetic-foot-wounds-kill-millions-but-high-tech-solutions-and-teamwork-are-making-a-difference-127218.

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Diabetic foot wounds kill millions, but high-tech solutions and teamwork are making a difference - Thehour.com

Intermountain to open new center for pediatric precision medicine – Healthcare IT News

Intermountain Primary Children's Hospital, along with University of Utah Health, and Intermountain Precision Genomics are teaming up to launch a pediatric center for personalized medicine that will serve the Intermountain West.

WHY IT MATTERS The center will use precision genomics to discover, address and treat genetic diseases, many of which affect infants and children and can cause life-long disability.

The Center will focus on precision diagnosis, gene therapies and novel therapeutics, and stem cell, immunologic and regenerative medicine.

Precision medicine includes applications across diagnostics, prevention and screening that takes into account individual variabilities in genes, environment, and lifestyle for every individual.

Through its work on precision diagnosis, the Center hopes to provide more targeted care to critically-ill children based on their genetic make-up, where rapid whole genome sequencing can quickly identify genetic causes of hard-to-diagnose diseases.

The initial efforts will be focused on providing answers to critically ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.

The research into gene therapies and novel therapeutics will help enable children with previously debilitating and fatal genetic diseases, with clinical trials testing gene therapy treatments for Duchenne's Muscular Dystrophy, Adrenoleukodystrophy, and other serious diseases.

The Center is also developing novel therapeutics that target specific diseases and improve health, with a release noting the Center is one of only six hospitals nationwide to provide gene therapy for the common childhood genetic condition spinal muscular atrophy.

Stem cell research uses a child's own cells, or genetically modifies a child's cells and immune system, to fight disease and promote healing, with additional research aimed at developing immunotherapy as a tool to fight pediatric brain tumors.

The organization also noted clinical trials are testing the use of stem cells in repairing diseased hearts and other tissues.

THE LARGER TRENDIntermountain has been busy on this front recently. In June, the health system announced that it is performing a massive clinical DNA study, pairing 500,000 samples drawn from Intermountain Healths patient population and analyzing them with help from deCODE, a subsidiary of Reykjavik-based Amgen.

"Better health and being able to cure common diseases is the promise of precision medicine, but its not happening fast enough," said Dr. Marc Harrison, president and CEO at Intermountain Healthcare, announcing that initiative. "For too long, the genetic code to better health has been locked. This collaboration with deCODE unlocks that insight so we can rapidly advance well-being not only for ourselves and our families, but for generations to come.

Intermountain's new pediatrics personalized medicine announcement also follows Mount Sinai's just-announced plans to build new precision medicine supercomputer, which will have 15 terabytes of memory, 14 petabytes of raw storage and a peak speed of 220 teraflops per second, to manage massive amounts of genomic data.

ON THE RECORD"Our mission is to leverage the expertise of our scientists, the clinical care of our physicians and care-givers, and the dedication of our community, to discover and develop new cures for children," said Dr. Josh Bonkowsky, Intermountain's medical director of the Primary Children's Center for Personalized Medicine, in a statement. "The work we are doing here and now is transforming pediatric medicine. We will not be done until we have put these diseases out of business."

Nathan Eddy is a healthcare and technology freelancer based in Berlin.Email the writer:nathaneddy@gmail.comTwitter:@dropdeaded209

Healthcare IT News is a publication of HIMSS Media.

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Intermountain to open new center for pediatric precision medicine - Healthcare IT News

Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know… – YourObserver.com

Regenerative Medicine and Stem Cell technologies... All Hype? Or The Future of Anti-Aging?

We have entered into the rapidly evolving age of Regenerative Medicine, using breakthroughs in cell to cell communication to reset your body's natural ability to heal and repair itself. Advances in Stem Cell technology race forward at an ever increasing rate as people just like you are demanding non-surgical alternatives to the multiple degenerative conditions of aging and inflammation.

If you are asking What is a Stem Cell?

Think of them as simply the master cells of rebuilding the body. They are the building blocks of our genetic code and cellular programming that coordinate healing through bio-signaling to restore our innate capacity of repair & regeneration.

Regenerative Medicine is the vanguard of 21st century health According to Journal: American College of Cardiology

Regenerative Aesthetics is a new field of regenerative medicine that aims to restore and renew the body at the cellular level, dramatically reversing the sands of time and maintaining an aesthetically desirable youthful appearance.

What we really now know is that men and women all over the world want their hair back, they want their sexual organs to work and they want to look and feel their best into their later years.

This brings us to the 3 Regenerative Therapies you should know about.

The first and most widely known of these regenerative therapies is Platelet Rich Plasma or PRP which utilizes your own Blood Plasma and Platelets in a concentrated form to activate the healing cascade. Recruiting your own innate Stem Cells for accelerated wound healing and tissue regeneration.

Known as Liquid Gold, your platelets and plasma have been shown to rejuvenate the

Unfortunately, we are finding clinically that not all PRP is created equally. In fact, some people have a very low concentration of these regenerative growth factors or a high amount of inflammatory cytokines resulting in inconsistencies from person to person, session to session. As PRP continues to get more popular in the mainstream, we find it important to share some of the newer, more optimal Regenerative Technologies that have been emerging.

Which leads us to... The second regenerative therapy you should know about.

Stem Cell Growth factors & Cytokines. Sourced from Bone Marrow Mesenchymal Stem Cells (MSC's) however, they do not contain any actual stem cells or DNA. Growth factors are naturally occurring proteins in your body that regulate cellular growth, healing, proliferation and differentiation under controlled conditions and play a role in cellular communication. They are master bio-signals acting as command and control over your body's natural healing processes and modulation of inflammation.

It has been shown that cells in aging skin generate less growth factors than cells in youthful skin. For example, by the time you are 50, on average 4% of these regenerative bone marrow MSCs are in circulation compared to what you had when you were in your teens. Hence, we age because we damage faster than we repair in our later years.

By simply adding concentrated MSC Growth factors & Cytokines to our Regenerative Therapies we can consistently improve hair loss, skin rejuvenation, collagen growth, sexual organ function, and more. We know for a fact that daily use of skin care products containing stable growth factors and cytokines help reduce the appearance of fine lines & wrinkles and improve skin tone & texture.

Lastly, and most importantly is the latest frontier and the 3rd regenerative therapy you should know about.

The Future of Regenerative Medicine... Known as Stem Cell Exosomes. Science is showing us that the optimal way to provide true stem cell therapy is to directly provide the cell bio-signals in high concentrations. After all, the signaling is what we really require to regenerate a normal healthy physiology.

Exosomes are regarded as the purest form of cellular therapy available today, providing a safe and anti-inflammatory environment for healing and repairing.

The AABB recently reported that up to one in three people in the U.S. could benefit from regenerative medicine.

At Rejuvenate 528 Regenerative Aesthetics Medical Spa, we can include exosomes for enhanced wellness to the majority of our Regenerative Aesthetic Services. This can benefit your overall health and vitality as this is reversing challenges of Inflammation!

Beauty radiates and vibrates at different frequencies in everyone I see! We love to uplift and Rejuvenate both the inner vitality and the outer Radiance of all of our patients and clients. They come for the personal attention and integrative approach using regenerative medicine modalities with ancient technologies. PA Sheri Suiter

Tap into your own healing potential with these types of bio-hacking technologies to enhance your regenerative potential and get the results you truly desire. Live longer, stronger and younger.

Book a consultation for the following Regenerative procedures:

*Medical Microneedling*Vampire Facial*Liquid Facelift*Breastlift*O-Shot*P-Shot*Penile Enhancement*Hair Restoration*Stretch mark & scar repair*Hand Rejuvenation*Joint Inflammation*Overall Vitality & Wellness

Sheri Suiter CLT, MS, PA-C, Founder of Rejuvenate 528 Regenerative Aesthetics Medical Spa in Sarasota, FLRejuvenate528.com

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Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know... - YourObserver.com

Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in…

BEDFORD, Mass., Nov. 26, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a peer-reviewed publication demonstrating its proprietary adeno-associated viral vectors (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in non-human primates (NHPs), highlighting their potential to deliver gene therapy for central and peripheral nervous system disorders.

The publication includes the initial characterization of biodistribution with three of Homologys 15 AAVHSCs, including their ability to transduce, or target, key cells following a single intravenous (I.V.) administration in NHPs. AAVHSCs are naturally occurring vectors originally isolated from human hematopoietic stem cells.

Many neurological diseases, including lysosomal storage and neuromuscular disorders, have cognitive and systemic components requiring a genetic medicine to reach multiple tissues to target the disease-relevant cell types, said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Here we evaluated the ability of three of our novel AAVHSCs to cross the blood-brain-barrier and the blood-nerve barrier after I.V. administration in NHPs in addition to other key tissues, which allows us to choose the vectors best suited for particular diseases. We have observed that small sequence changes among our family of AAVHSCs are associated with differences in their ability to target disease-relevant tissues. We continue to characterize these properties and the potential of AAVHSCs as vehicles for therapeutic delivery.

Following I.V. administration of AAVHSC -7, -15 and -17 in NHPs, analyses showed transduction and transgene expression:

The publication, Clade F AAVHSCs Cross the Blood Brain Barrier and Transduce the Central Nervous System in Addition to Peripheral Tissues Following Intravenous Administration in Nonhuman Primates, was peer-reviewed and published in the journal PLOS ONE. For more information, please visithttps://journals.plos.org/plosone/article?id=10.1371/journal.pone.0225582or http://www.homologymedicines.com/publications.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; beliefs about preclinical data and the properties and potential of our AAVHSCs; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter endedSeptember 30, 2019and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in...

Worldwide Markets for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing, Forecast to 2030 – Robust Pipeline of Therapy Candidates and…

Dublin, Nov. 27, 2019 (GLOBE NEWSWIRE) -- The "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors)" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the rapidly growing market of viral and non-viral vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies with in-house manufacturing facilities. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe.

At present, 10+ genetically modified therapies have received approval / conditional approval in various regions of the world; these include (in the reverse chronological order of year of approval) Zynteglo (2019), Zolgensma (2019), Collategene (2019), LUXTURNA (2017), YESCARTA (2017), Kymriah (2017), INVOSSA (2017), Zalmoxis (2016), Strimvelis (2016), Imlygic (2015), Neovasculagen (2011), Rexin-G (2007), Oncorine (2005) and Gendicine (2003). In addition, over 500 therapy candidates are being investigated across different stages of development. The growing number of gene-based therapies, coupled to their rapid progression through the drug development process, has created significant opportunities for companies with expertise in vector manufacturing.

Presently, a number of industry (including both well-established companies and smaller R&D-focused initiatives), and non-industry players (academic institutes) claim to be capable of manufacturing different types of viral and non-viral vectors. In addition, there are several players offering novel technology solutions, which are capable of improving existing genetically modified therapy products and upgrading their affiliated manufacturing processes.

Considering prevalent and anticipated future trends, we believe that the vector and gene therapy manufacturing market is poised to grow steadily, driven by a robust pipeline of therapy candidates and technical advances aimed at mitigating existing challenges related to gene delivery vector and advanced therapy medicinal products.

Chapter Outlines

Chapter 2 is an executive summary of the insights captured in our research. The summary offers a high-level view on the likely evolution of the vector and gene therapy manufacturing market in the short to mid-term, and long term.

Chapter 3 is a general introduction to the various types of viral and non-viral vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vehicles. The chapter also provides a brief description of the clinical and approved pipeline of genetically modified therapies. Further, it includes a review of the latest trends and innovations in the contemporary vector manufacturing market.

Chapter 4 provides a detailed overview of around 80 companies, featuring both contract service providers and in-house manufacturers that are actively involved in the production of viral vectors and / or gene therapies utilizing viral vectors. The chapter provides details on the year of establishment, scale of production, type of viral vectors manufactured (AAV, adenoviral, lentiviral, retroviral and others), location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of production (fulfilling in-house requirements / for contract services).

Chapter 5 provides an overview of around 30 industry players that are actively involved in the production of plasmid DNA and other non-viral vectors and / or gene therapies utilizing non-viral vectors. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 6 provides an overview of around 80 non-industry players (academia and research institutes) that are actively involved in the production of vectors (both viral and non-viral) and / or gene therapies. The chapter provides details on the year of establishment, scale of production, location of manufacturing facilities, type of vectors manufactured (AAV, adenoviral, lentiviral, retroviral, plasmid DNA and others), applications of vectors (gene therapies, cell therapies, vaccines and others) and purpose of vector production (fulfilling in-house requirements / for contract services).

Chapter 7 features detailed profiles of the US-based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience and an informed future outlook.

Chapter 8 features detailed profiles of EU based contract service providers / in-house manufacturers that possess commercial-scale capacities for the production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 9 features detailed profiles of Asia-Pacific based contract service provider(s) / in-house manufacturer(s) that possess commercial scale capacities for production of viral vectors/plasmid DNA. Each profile presents a brief overview of the company, its financial information (if available), details on vector manufacturing facilities, manufacturing experience, and an informed future outlook.

Chapter 10 provides detailed information on other viral / non-viral vectors (including alphavirus vectors, Bifidobacterium longum vectors, Listeria monocytogenes vectors, myxoma virus-based vectors, Sendai virus-based vectors, self-complementary vectors (improved versions of AAV), and minicircle DNA and Sleeping Beauty transposon vectors (non-viral gene delivery approach)) that are currently being utilized by pharmaceutical players to develop gene therapies, T-cell therapies and certain vaccines, as well. This chapter presents overview on all the aforementioned types of vectors, along with examples of companies that use them in their proprietary products. It also includes examples of companies that are utilizing specific technology platforms for the development/manufacturing of some of these novel vectors.

Chapter 11 features an elaborate analysis and discussion of the various collaborations and partnerships related to the manufacturing of vectors or gene therapies, which have been inked amongst players. It includes a brief description of the purpose of the partnership models (including licensing agreements, mergers/acquisitions, product development, service alliances, manufacturing, and others) that have been adopted by the stakeholders in this domain, since 2015. It consists of a schematic representation showcasing the players that have forged the maximum number of alliances. Furthermore, we have provided a world map representation of the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 12 presents a collection of key insights derived from the study. It includes a grid analysis, highlighting the distribution of viral vectors and plasmid DNA manufacturers on the basis of their scale of production and purpose of manufacturing (fulfilling in-house requirement/contract service provider). In addition, it consists of a logo landscape, representing the distribution of viral vector and plasmid DNA manufacturers based on the type of organization (industry / non-industry) and size of employee base. The chapter also consists of six world map representations of manufacturers of viral / non-viral vectors (lentiviral, adenoviral, AAV and retroviral vectors, and plasmid DNA), depicting the most active geographies in terms of the presence of the organizations. Furthermore, we have provided a schematic world map representation to highlight the locations of global vector manufacturing hubs across different continents.

Chapter 13 highlights our views on the various factors that may be taken into consideration while pricing viral vectors/plasmid DNA. It features discussions on different pricing models/approaches that manufacturers may choose to adopt to decide the prices of their proprietary products.

Chapter 14 features an informed estimate of the annual demand for viral and non-viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies. This section offers an opinion on the required scale of supply (in terms of vector manufacturing services) in this market. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analysed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.

Chapter 15 features an informed analysis of the overall installed capacity of the vectors and gene therapy manufacturers. The analysis is based on meticulously collected data (via both secondary and primary research) on reported capacities of various small-sized, mid-sized and large companies, distributed across their respective facilities. The results of this analysis were used to establish an informed opinion on the vector production capabilities of the organizations across different types of vectors (viral vectors, plasmid DNA, and both), scale of operation (clinical and commercial) and geographies (North America, EU, Asia-Pacific and the rest of the world).

Chapter 16 presents a comprehensive market forecast analysis, highlighting the likely growth of vector and gene therapy manufacturing market till the year 2030. We have segmented the financial opportunity on the basis of [A] type of vectors (AAV vector, adenoviral vector, lentiviral vector, retroviral vector, plasmid DNA and others), [B] applications (gene therapy, cell therapy and vaccines), [C] therapeutic area (oncological disorders, inflammation & immunological diseases, neurological disorders, ophthalmic disorders, muscle disorders, metabolic disorders, cardiovascular disorders and others), [D] scale of operation (preclinical, clinical and commercial) and [E] geography (North America, Europe, Asia Pacific and rest of the world). Due to the uncertain nature of the market, we have presented three different growth tracks outlined as the conservative, base and optimistic scenarios.

Chapter 17 provides details on the various factors associated with popular viral vectors and plasmid DNA that act as market drivers and the various challenges associated with the production process. This information has been validated by soliciting the opinions of several industry stakeholders active in this domain.

Chapter 18 presents insights from the survey conducted on over 160 stakeholders involved in the development of different types of gene therapy vectors. The participants, who were primarily Director / CXO level representatives of their respective companies, helped us develop a deeper understanding on the nature of their services and the associated commercial potential.

Chapter 19 summarizes the entire report. The chapter presents a list of key takeaways and offers our independent opinion on the current market scenario and evolutionary trends that are likely to determine the future of this segment of the industry.

Chapter 20 is a collection of transcripts of the interviews conducted with representatives from renowned organizations that are engaged in the vector and gene therapy manufacturing domain. In this study, we spoke to Menzo Havenga (Chief Executive Officer and President, Batavia Biosciences), Nicole Faust (Chief Executive Officer & Chief Scientific Officer, CEVEC Pharmaceuticals), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences), Olivier Boisteau, (Co-Founder / President, Clean Cells) and Xavier Leclerc (Head of Gene Therapy, Clean Cells), Laurent Ciavatti (Business Development Manager, Clean Cells), Joost van den Berg (Director, Amsterdam BioTherapeutics Unit), Bakhos A Tannous (Director, MGH Viral Vector Development Facility, Massachusetts General Hospital), Colin Lee Novick (Managing Director, CJ Partners), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Semyon Rubinchik (Scientific Director, ACGT), Alain Lamproye (President of Biopharma Business Unit, Novasep), Astrid Brammer (Senior Manager Business Development, Richter-Helm), Brain M Dattilo (Business Development Manager, Waisman Biomanufacturing), Marco Schmeer (Project Manager, Plasmid Factory) and Tatjana Buchholz (Marketing Manager, Plasmid Factory), and Nicolas Grandchamp (R&D Leader, GEG Tech).

Chapter 21 is an appendix, which provides tabulated data and numbers for all the figures in the report.

Chapter 22 is an appendix that provides the list of companies and organizations that have been mentioned in the report.

Key Topics Covered

1. Preface2. Executive Summary3. Introduction4. Viral Vector and Gene Therapy Manufacturers (Industry Players): Competitive Landscape5. Plasmid DNA and Gene Therapy Manufacturers (Industry Players): Competitive Landscape6. Vector and Gene Therapy Manufacturers (Non-Industry Players): Competitive Landscape7. Vector and Gene Therapy Manufacturers in North America8. Vector and Gene Therapy Manufacturers in Europe9. Vector and Gene Therapy Manufacturers in Asia-Pacific10. Emerging Vectors11. Recent Collaborations and Partnerships12. Key Insights13. Viral Vector and Plasmid DNA Cost Price Analysis14. Capacity Analysis15. Demand Analysis16. Market Sizing and Opportunity Analysis17. Key Drivers and Challenges18. Survey Analysis19. Concluding Remarks20. Executive Insights21. Appendix I: Tabulated Data22. Appendix II: List of Companies and Organizations

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/4m9jtt

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Worldwide Markets for Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing, Forecast to 2030 - Robust Pipeline of Therapy Candidates and...

Inside the Beltway: Abortion, immigration among forbidden topics at Thanksgiving table – Washington Times

The warnings are already up in the popular press: Conversations during the Thanksgiving feast can be hazardous if they veer into political territory. But political talk can take place in theory: A data research company has now determined what topics are safe to talk about on the holiday.

With some qualitative analysis and a little common sense, weve created a cheat sheet that will help you blaze a path through Thanksgiving dinner that steers clear of treacherous political pitfalls and dangerous inter-uncle conflicts, reports Ranker.com, a Los Angeles-based media company which uses crowdsourcing to rank public opinion on multiple topics, typically at the rate of 15 million votes a month.

They have determined what political topics are the least and the most likely to set off a Thanksgiving dinner squabble. Their judgment is based on 300,000 votes from 40,000 respondents.

The topics to avoid this year: Abortion, immigration, terrorism and gender equality. The topics which are safe for dinnertime discussion: Ineffective government, health care reform and education.

If there are millennials present, they will be triggered, the organization says, by talk of abortion, police brutality and pollution. Generation X members will be set off by such topics as homelessness, affordable housing and campaign finance reform. Baby boomers will go to battle over terrorism, immigration and the moral decline of the nation.

The organization also has warnings for dinner hosts in certain states. If they live in Florida, their guests will be particularly sensitive about discussions of vaccines. In Indiana, its gender equality while Georgia diners are prone to fight over police brutality. Beware of talking about gun control at dinner tables in both California and Missouri; Texans get feisty over moral decline. New Yorkers get upset over transgender issues.

We examined each issue on a case-by-case basis to find the topics that are most likely to cause disagreement, as well as the ones on which people tend to either agree or not care about, Ranker.com explains.

A VERY SPECIALIZED MEAL

While most of us are enjoying turkey and pumpkin pie on Thanksgiving, the staff at one laboratory at Cedars-Sinai Medical Center in Los Angeles will be busy serving a meal to stem cells.

Stem cells do not observe national holidays, says Loren Ornelas-Menendez, manager of the very specialized lab that converts samples of adult skin and blood cells into stem cells which the human body uses to make our cells in the first place.

These special cells help medical scientists learn how diseases develop and how they might be cured. The lab is tending millions of them. Oh, but they have needs.

Stem cells are living creatures that must be hand-fed a special formula each day, monitored for defects and maintained at just the right temperature. And that means the cell lab is staffed every day, 52 weeks a year, the lab notes in a public advisory.

Many people have dogs. We have stem cells, says Ms. Ornelas-Menendez.

Derived from hundreds of healthy donors and patients, the resident induced pluripotent stem cells or iSPCs are keys to potential treatments for diabetes, breast cancer, Alzheimers disease, blindness, Parkinsons disease and Crohns disease, among other conditions. Ten lab technicians monitor the cells through microscopes each day and cull out any cells which have gone awry for one reason or another.

But what do they eat even on Thanksgiving?

While the cells get sorted, a special feeding formula is defrosting in a dozen bottles spread around a lab bench. The formula includes sodium, glucose, vitamins and proteins. Using pipettes, employees squeeze the liquid into food wells inside little compartments that contain the iPSCs. Afterward, they return the cells to their incubators, the lab advises.

Lab director Dhruv Sareen suggests that people consider offering a toast to the stem cells on Thanksgiving.

One day the cells they tend could lead to treatments for diseases that have plagued humankind for centuries, he says. And thats something to be truly thankful for.

THE GIPPERS FAVORITE

Back by popular demand, Inside the Beltway again shares this little known but historic recipe for President Reagans Favorite Macaroni and Cheese enjoyed by Ronald Reagan and his family on Thanksgiving and other holidays. What follows is a step-by-step shared by Mrs. Ronald Reagan, Washington, D.C., Wife of the President in a spiral-bound community cookbook published by the American Cancer Societys Northern Virginia division in 1983. The recipe serves six and is baked at 350 degrees F for 45 minutes.

The directions are from the cookbook reflecting the style, perhaps, of another era:

1/2 pound macaroni, 1 teaspoon butter, 1 egg, beaten; 1 teaspoon salt, 1 teaspoon dry mustard, 3 cups grated cheese, sharp; 1 cup milk.

Boil macaroni in water until tender and drain thoroughly. Stir in butter and egg. Mix mustard and salt with 1 tablespoon hot water and add to milk. Add cheese leaving enough to sprinkle on top. Pour into buttered casserole, add milk, sprinkle with cheese. Bake until custard is set and top is crusty.

Curious about what transpired at a Reagan Thanksgiving? A 1985 Los Angeles Times account noted this:

President and Mrs. Reagan gathered with their family for a quiet Thanksgiving dinner at their fogbound ranch in the Santa Ynez mountains, where the main topic of conversation was the weather. The Reagans did not seem to mind the enforced seclusion as they sat down to a traditional turkey dinner, prepared by Ann Allman, the Reagan familys longtime cook in California. It was an all-American menu that included cornbread dressing, cranberries, string beans, mashed potatoes, salad, pumpkin pie and monkey bread, a family favorite.

POLL DU JOUR

46% of Americans say long standing family tensions are the cause of family fights during holidays.

37% say general politics is the cause; 33% cite the 2020 presidential race.

24% say someones future plans cause the fights; 24% say money.

22% say the behavior of guests; 21% say drinking and alcohol.

18% say holiday cooking is the cause.

Source: A YouGov poll of 1,310 U.S. Adults conducted Sept. 25-26 and released Tuesday.

Have a happy Thanksgiving and thank you for reading Inside the Beltway.

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Inside the Beltway: Abortion, immigration among forbidden topics at Thanksgiving table - Washington Times

BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND – BioSpace

BrainStorms Chief Operating and Chief Medical Officer, Ralph Kern MD MHSc, will make a podium presentation at Clinical Trials Session

NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.

The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.

Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.

Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

BRAINSTORM CONTACTS:

Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839Email: sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - BioSpace

Canine Stem Cell Therapy Market Value Projected to Expand by 2026 – The Hilltop Monitor

The current Stem Cells Cryopreservation Equipments market research report has demonstrated all the vital market growth factors and economic fluctuations mentioned owing to the vast attention gained in recent years.

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Common sense to rest Man United seniors for Astana trip: SolskjaerA statement released by the club In February said "everybody looks forward to seeing him wear the red shirt again". Their last outing against Sheffield United ended in 3-3 draw and they surely don't repeat that feat this time.

Global Market portal aims to provide reports like these in order to draw the attention of numerous clients wanting to extrapolate some of the vital details of the Stem Cells Cryopreservation Equipments market on a global scale. The report provides an extensive evaluation of each market segment considering its demand, production, sales revenue, and potential growth. The solid research on the Animal Stem Cell Therapy Market is prepared with the aim to meet the requirements of the customer in terms of the availability of data, analytics, statistics, and an accurate forecast market.The market report also presents the landscape and a corresponding detailed analysis of the major players operating in the market. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length. USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits.

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- Who are the key market players and what are their strategies in the global Animal Stem Cell Therapy market? Moreover, studies showing the excellent level of safety granted by allogeneic stem cell therapy are also rising in numbers, leading to increased confidence among pet owners as well as veterinarians regarding canine stem cell therapy.

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Canine Stem Cell Therapy Market Value Projected to Expand by 2026 - The Hilltop Monitor

Global Stem Cells Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 – Industry News Info

The Global Stem Cells Market report study includes an elaborative summary of the Stem Cells market that provides in-depth knowledge of various different segmentations. Stem Cells Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis of Stem Cells Market. The information includes the company profile, annual turnover, the types of products and services they provide, income generation, which provide direction to businesses to take important steps. Stem Cells delivers pin point analysis of varying competition dynamics and keeps ahead of Stem Cells competitors such as CCBC, Vcanbio, Boyalife, Beikebiotech.

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The main objective of the Stem Cells report is to guide the user to understand the Stem Cells market in terms of its definition, classification, Stem Cells market potential, latest trends, and the challenges that the Stem Cells market is facing. In-depth researches and Stem Cells studies were done while preparing the Stem Cells report. The Stem Cells readers will find this report very beneficial in understanding the Stem Cells market in detailed. The aspects and information are represented in the Stem Cells report using figures, bar-graphs, pie diagrams, and other visual representations. This intensifies the Stem Cells pictorial representation and also helps in getting the Stem Cells industry facts much better.

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Global Stem Cells Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 - Industry News Info

Lulu and Nana are the result of a defiant experiment in human gene modification – The Irish Times

Remember these two names, Lulu and Nana. They are twin girls born in China in October 2018, and we already know they are going to be famous.

They are the worlds first genetically-edited humans, and their progress through life will be monitored intensively by medical researchers over the coming years.

Dont doubt their future celebrity. Remember the headlines when Dolly the sheep arrived in 1996, the worlds first mammal cloned directly from an adult cell. Or how about Louise Brown, who made headlines in 1978 as the first baby to be conceived using invitro fertilisation (IVF) techniques.

Although the methods used to bring about these three births are light years apart, all three involve the delivery of an offspring using unorthodox methods.

All three were also controversial in their day. There was no end of condemnation and criticism about playing god and defying the laws of nature for the first two, but now IVF is commonplace for lots of medical reasons and is accepted as a standard medical practice. And news of another species successfully cloned in the lab would not make headlines today.

However, the genetic modification of the twins is a different matter, a genetic change introduced before birth to deliver a permanent alteration of their original genome that will be passed down from generation to generation.

Prof He Jiankui of the Southern University of Science and Technology carried out the genetic change needed to permanently modify the twins genome. He introduced a mutation that gave Lulu and Nana resistance to the Human Immunodeficiency Virus, the virus that causes Aids, and revealed his successful genetic modification one year ago this month.

The backlash was immediate and severe. There was international condemnation that China allowed experimentation on humans. Last January his university sacked him.

The scientific community also criticised the work as having crossed an important red line for genetics research, the reality that we still know too little about how even the smallest genetic change might have unexpected impacts downstream in other parts of the genome.

It was bad enough that a modification had taken place, but the modification was in the germline, the cells that bring about the next generation and the next and the next.

Perhaps the lure of notoriety proved too strong or the desire to be the first, but He Jiankui crossed that line, helped along the way through use of a gene-editing method known as CRISPR-Cas9.

This method emerged over several years, and has become the gene-cutting tool of choice because it allows very tight control over how a gene can be modified, added to or deleted.

When it came into widespread use the scientific community recognised immediately that controls on its application were necessary to prevent its use in human gene modification. This should have prevented He Jiankui from attempting such a daring and defiant experiment but it didnt, and Lulu and Nana were the result.

They were born healthy but now scientists will want to know whether there are unexpected or unwanted effects or other issues that arise. Already researchers have raised doubts about the gene modification that confers resistance to HIV, which means some level of susceptibility to the virus may remain.

The modified gene, known as CCR5, also has other roles in the body, and its modified actions may affect the lifespan of the twins. One large study involving 410,000 subjects showed that people with a similar mutated version of CCR5 were 20 per cent more likely to die before reaching the age of 78.

Despite these misgivings there is no doubt that CRISPR-Cas9 will in the future be used to modify the human genome in the battle against difficult diseases such as cancer and in genetic disorders caused by specific gene mutations. The tool is far too important to avoid its eventual use, or the use of some other similar but as yet unidentified gene-cutting method.

Similarly there is little doubt that online charlatans will offer promised but undeliverable cures using CRISPR-Cas9, as was the case with earlier technologies such as stem cells.

Lulu and Nana, meanwhile, will get on with living their lives, doing what babies do. Their names are pseudonyms in an attempt to conceal them from public view, but it is likely that we may learn their real names in the future.

Lulu and Nanas story came to mind in light of research published last week (November 21st) in the journal Cell about how attempting to create designer babies using other advanced technology could still remain a costly waste of time.

It involves choosing an embryo based on its potential to be tall or smarter than average, but accomplishing this via something like CRISPR-Cas9 is too far beyond our current abilities. Instead the international team of scientists set up a model to simulate one method called pre-implantation genetic testing.

This involves screening the genome for genes that have an association to a given trait, in this studys case intelligence and height, and giving them a score. The team found, however, that at best the top-scoring embryo might be expected to be 2.5cm taller than average and at best 2.5 IQ points above average.

Lurking behind all of this remains the most challenging of issues, the ethics surrounding the use of this technology.

As usual the ethics questions that should have been asked first are the ones obscured by the advance of these promising discoveries.

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Lulu and Nana are the result of a defiant experiment in human gene modification - The Irish Times