Duke researchers land $6M in federal grants to advance gene editing – WRAL Tech Wire

DURHAM Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the source but significant hurdles must be overcome before genome-editing treatments are ready for use in humans.

Enter the National Institutes of Health Common FundsSomatic Cell Genome Editing (SCGE)program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.

Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.

In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.

Duke engineers improve CRISPR genome editing with biomedical tails

There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.

For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.

Duke researchers: Single CRISPR treatment provides long-term benefits in mice

Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.

Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.

Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing bothnew CRISPR systems for gene regulationandto regulate the epigenome rather than permanently delete DNA sequences.

Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in usingAAV and CRISPR to treat animal models of DMD.

We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.

Click through to learn more about theDuke Center for Advanced Genomic Technologies.

(C) Duke University

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Duke researchers land $6M in federal grants to advance gene editing - WRAL Tech Wire

TEDCO funds open again for applications – Maryland Daily Record

Four Maryland Technology Development Corporation funds will begin accepting applications for investments again after they had delayed investments while adapting regulations to an oversight law passed by the Maryland General Assembly last year.

The Builder Fund, the Maryland Venture Fund, the Rural Business Innovation Initiative and the Seed Fund are now accepting applications for investment.

Stephen Auvil (File photo)

We are thrilled to bring these application portals live after the team has worked hard to finalize the regulations, Stephen Auvil, TEDCOs executive vice president, said in a statement. Its our goal to get back to what TEDCO does best, and thats building great, Maryland-based startups and continuing to grow innovation and entrepreneurship in the state.

A legislative audit issued last February found issues that included how TEDCO directed funding to companies that were not primarily based in Maryland and invested in companies that had associations with the Maryland Venture Funds advisory committee.

In the aftermath of the audit, lawmakers put more restrictions in place to govern how TEDCO invests its money and what oversights are in place to govern the agency.

One of the lawmakers requirements was that TEDCO develop an application process for its investment programs. That application process is now ready, TEDCO said Friday.

The Builder Fund is designed to help startups founded by people from economically disadvantaged background. The Rural Business Innovation Initiative invests in companies from rural Maryland. The Maryland Venture Fund is a venture capital fund focused on growth stage companies, and the Seed Fund invests in seed-stage companies.

TEDCO explained to lawmakers last month how it had crafted its regulations to comply with the oversight law.

The new regulations more clearly define what a Maryland business is and how it can qualify for investments. They also define how a company not defined as a Maryland business can receive investment if it will have a substantial economic impact on the state.

Under the new regulations, state businesses must have their principal base of operations in Maryland; have more than half of their workforce in Maryland; and intend to maintain their base of operations in Maryland.

TEDCO also has created provisions that would allow it to claw back its investment if a company leaves the state.

While TEDCO is accepting applications now, investments could still take a little while to happen. The agencys Maryland Venture Fund Authority does not yet have a quorum of members appointed to disburse funds.

The Maryland Venture Fund will also only be accepting applications from current Maryland Venture Fund portfolio companies. It anticipates accepting applications from other companies for the fund later this spring.

TEDCO operates eight funds. Programs like the Maryland Innovation Initiative, which helps commercialize university technology, and the Maryland Stem Cell Research Fund were not affected by the new regulations.

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TEDCO funds open again for applications - Maryland Daily Record

Cell Culture Protein Surface Coating Market Competitive Environment and Higher Growth Rate with Forecast to 2025 – Filmi Baba

Global Cell Culture Protein Surface Coating Market: Snapshot

The global market for cell culture protein surface coatings is slated to expand at a highly promising pace in the next few years, thanks to the vast rise in investments by governments and market players in stem cell research and development activities. Cell culturing is a method used for growing artificial living cells outside the natural environment, under controlled physical conditions. These cells are used to develop model systems for study and research of cellular structures as well as for drug discovery and genetic engineering.

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Thus, the growing scope of cell cultures in various applications has led to the development of the 3D cell culture technique, which has been considered one of the key factors responsible for the overall past development of the cell culture protein surface coatings market. Earlier, only a meager percentage of researchers preferred using 3D cell culture technique for drug discovery. However, there has been a dynamic shift from the traditional methods to the current cell culture methods.

Moreover, commercial production of drugs and biologics such as proteins, antibodies, and vaccines using cell culture has helped expand the scope of the latter in the global market. Commercial production has provided extensive business opportunities to manufacturers in the global market. Diverse applications of stem cells such as development of bone grafts and artificial tissue are also expected to fuel the demand for cell culture protein surface coatings over the forecast period. In addition, increasing cell culture applications in toxicology studies and cell-based assays are further pushing the growth of the market.

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Global Cell Culture Protein Surface Coating Market: Overview

Cell culture protein surface coatings help in improving cell attachment, growth, and differentiation. They facilitate consistent performance in various cell-based assays and in-vitro culture by improving cell adhesion. A variety of adhesion proteins and other biological materials derived from various sources are being used to enhance performance in cell culture, especially in cell lines that are hard to attach, such as transfected cells. The major types of cell culture are animal-derived protein, human-derived protein, synthetic protein, and plant-derived protein. Good cell attachment has gained increased significance in recent years for improving the recovery of cells from frozen cultures and increasing the stability of attached surfaces. With constant advances in stem cell therapies, a number of advanced protein surface coatings have emerged to study stem cells and to further the potential of regenerative medicine. These developments have positively affected the growth of the global cell culture protein surface coating market.

Global Cell Culture Protein Surface Coating Market: Key Trends

The increasing focus of numerous biotechnology companies and research laboratories on stem cell research to develop therapies for a range of chronic diseases is a key factor propelling the cell culture protein market. Considerable investment by the governments of various countries to fund several R&D activities related to regenerative medicine has fuelled the market. Coupled with this, the rising demand for biopharmaceutical products such as antibodies, vaccines, and drugs has stimulated the demand for cell culture protein surface coatings. The growing research on stem cells for finding therapies for various cardiovascular and neurological diseases is expected to boost the market in the coming years. The growing prominence of 3D cell culture over 2D cell cultures is expected to unlock exciting opportunities in the cell culture protein surface coating market.

Global Cell Culture Protein Surface Coating Market: Market Potential

The American Heart Association (AHA), together with the Paul G. Allen Frontiers Group, announced in April, 2017 two grantseach worth US$1.5 millionto scientists working on cardiovascular extracellular matrix (ECM) research. Interested researchers have to apply for grants by May 10, and each of the two winners will be entitled to the magnanimous sum.

The ECM regulates all vital cell functions and is considered a highly useful biomaterial for investigators. This can be applied as a stable coating to be used in a variety of cell cultures. The initiative focused on investigating the role of ECM in the initiation and progression of a number of cardiovascular diseases, such as hypertensive heart disease, ischemic heart disease, cardiomyopathies, congenital cardiovascular malformations, and atherosclerosis and vascular diseases. The funding will further the investigation into the diagnosis, prevention, and treatment of cardiovascular diseases. One of the most commonly used protein surface coatings used in ECM is collagen, which facilitates cell adherence, growth, migration, differentiation, and proliferation. The major research initiatives, opine the AHA, will be greatly useful in setting up a new paradigm in research in cell structure in biosciences.

Global Cell Culture Protein Surface Coating Market: Regional Outlook

North America is a prominent market for cell culture protein surface coatings and is expected to exhibit significant growth over the forecast period. The impressive growth in the regional market is attributed to the presence of a robust healthcare infrastructure and considerable advances in stem cell research. In addition, the soaring demand for regenerative medicines for a range of autoimmune therapies is expected to fuel the demand for surface coatings for improving the performance of in-vivo culture.

The Asia Pacific market for cell culture protein surface coating is poised to offer lucrative avenues for players in the market. Favorable regulations for biologics development and a burgeoning biotechnology industry are the factors expected to lead to substantial demand for cell culture protein surface coatings.

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Global Cell Culture Protein Surface Coating Market: Competitive Analysis

The market is fairly competitive due to the presence of a large number of regional and global vendors. Leading vendors are actively focused on providing solutions having cell attachment ability and promoting in-vitro cell functions for a variety of cell types to gain competitive edge over others. Leading players operating in this market include Sigma-Aldrich Corporation, Agilent Technologies, Thermo Fisher Scientific, EMD Millipore, Corning Incorporated, Biomedtech Laboratories Inc., Neuvitro Corporation, and Progen Biotechnik GmbH.

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Cell Culture Protein Surface Coating Market Competitive Environment and Higher Growth Rate with Forecast to 2025 - Filmi Baba

Human Embryonic Stem Cells | The Embryo Project Encyclopedia

Human Embryonic Stem Cells

Stem cells are undifferentiated cells that are capable of dividing for long periods of time and can give rise to specialized cells under particular conditions. Embryonic stem cells are a particular type of stem cell derived from embryos. According to US National Institutes of Health (NIH), in humans, the term embryo applies to a fertilized egg from the beginning of division up to the end of the eighth week of gestation, when the embryo becomes a fetus. Between fertilization and the eighth week of gestation, the embryo undergoes multiple cell divisions. At the eight-cell stage, roughly the third day of division, all eight cells are considered totipotent, which means the cell has the capability of becoming a fully developed human being. By day four, cells begin to separate and form a spherical layer which eventually becomes the placenta and tissue that support the development of the future fetus. A mass of about thirty cells, called the inner cell mass, forms at one end of the sphere and eventually becomes the body. When the sphere and inner cell mass are fully formed, around day 5, the pre-implantation embryo is referred to as a blastocyst. At this point the cells in the inner cell mass have not yet differentiated, but have the ability to develop into any specialized cell type that makes up the body. This property is known as pluripotency. As of 2009, embryonic stem cells refer to pluripotent cells that are generally derived from the inner cell mass of blastocysts.

In November 1998, two independent publications announced the first successful isolation and culture of pluripotent human stem cells. While working at the Wisconsin National Primate Research Center, located at the University of Wisconsin-Madison, James A. Thomson and his team of researchers cultured human embryonic stem cells from the inner cell mass of donated embryos originally produced for in vitro fertilization. The characteristics of the cultured cells were consistent with previously identified features in animal stem cells. They were capable of long-term self-renewal and thus could remain undifferentiated for long periods of time; they had particular surface markers; and they were able to maintain a normal and stable karyotype. Thomsons team also observed derivatives of all the three germ layersendoderm, mesoderm, and ectoderm. Since the three germ layers precede differentiation into all the cell types in the body, this observation suggested that the cultured cells were pluripotent. The team published Embryonic Stem Cell Lines Derived from Human Blastocysts, in the 6 November Science issue. Soon afterwards, a research team led by John D. Gearhart at the Johns Hopkins School of Medicine, published Derivation of Pluripotent Stem Cells from Cultured Human Primordial Germ Cells in Proceedings of the National Academy of Science. The paper detailed the process by which pluripotent stem cells were derived from gonadal ridges and mesenteries extracted from aborted five-to-nine week old human embryos. Gearhart and his team noted the same observations as Thomsons team. Despite coming from different sources, according to NIH, the resultant cells seem to be the same.

The largest source of blastocysts for stem cell research comes from in vitro fertilization (IVF) clinics. Used for reproductive purposes, IVF usually produces an abundance of viable blastocysts. Excess blastocysts are sometimes donated for research purposes after obtaining informed consent from donors. Another potential method for producing embryonic stem cells is somatic cell nuclear transfer (SCNT). This has been successfully done using animal cells. The nucleus of a differentiated adult cell, such as a skin cell, is removed and fused with an enucleated egg, an egg with the nucleus removed. The egg, now containing the genetic material from the skin cell, is believed to be totipotent and eventually develops into a blastocyst. As of mid-2006, attempts to produce human embryonic stem cells using SCNT have been unsuccessful. Nonetheless, scientists continue to pursue this method because of the medical and scientific implications of embryonic stem cells lines with an identical genetic makeup to particular patients. One problem faced in tissue transplants is immune rejection, where the host body attacks the introduced tissue. SCNT would be a way to overcome the incompatibility problem by using the patients own somatic cells.

Recent discoveries in cultivating human embryonic stem cells may potentially lead to major advancements in understanding human embryogenesis and medical treatments. Previously, limitations in access and environmental control have stunted research initiatives aimed at mapping out the developmental process. Insights into differentiation factors may lead to treatments into such areas as birth defects. Manipulation of the differentiation process may then lead to large supplies of stem cells for cell-based therapies on patients with Parkinsons disease, for example. In theory adult stem cells can also be cultivated for such purposes, but isolating and identifying adult stem cells has been difficult and the prospects for treatment are more limited than using embryonic stem cells.

Despite the potential benefits that may come about through human embryonic stem cell research, not everyone in the public embraces it. Several ethical debates surround this newly developing research field. Much of the debate stems from differing opinions on how we should view embryos: is an embryo a person? Should an embryo be considered property? Ethical concerns in embryonic stem cell research include destroying human blastocysts, laws surrounding informed consent, and particularly for SCNT, misapplication of techniques for reproductive cloning. For the latter concern, SCNT does produce a blastocyst which contains stem cell clones of an adult cell, but the desired application is in growing replacement tissues. Still, a portion of the public fears the hypothetical one day, when someone decides to use SCNT to develop and raise a human clone.

The public debate continues, advancing along with the changes in the field. As of 2006, public opinion polls showed that majority of religious and non-religious Americans now support embryonic stem cell research, but opinions remain divided over whether it is legitimate to create or use human blastocysts solely for research.

Wu, Ke, "Human Embryonic Stem Cells".

(2010-09-13). ISSN: 1940-5030 http://embryo.asu.edu/handle/10776/2055.

Arizona State University. School of Life Sciences. Center for Biology and Society. Embryo Project Encyclopedia.

Arizona Board of Regents Licensed as Creative Commons Attribution-NonCommercial-Share Alike 3.0 Unported (CC BY-NC-SA 3.0) http://creativecommons.org/licenses/by-nc-sa/3.0/

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Human Embryonic Stem Cells | The Embryo Project Encyclopedia

Embryonic stem cell | biology | Britannica

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Alternative Titles:ES cell, ESC

Embryonic stem cells (often referred to as ES cells) are stem cells that are derived from the inner cell mass of a mammalian embryo at a very early stage of development, when it is composed of a hollow sphere of dividing cells

In contrast, embryonic stem cells (ESCs) can be harvested once and cultured indefinitely. Moreover, ESCs are pluripotent, meaning that they can be directed to differentiate into any cell type, which makes them an ideal cell source for regenerative medicine.

condemned medical research using embryonic stem cells, though it endorsed research with adult stem cells. While many theologians, clergy, and laypersons agreed with church policy on these matters, many others disagreed and even chose to defy it.

which point a culture of embryonic stem cells (ESCs) can be created from the inner cell mass of the blastocyst. Mouse, monkey, and human ESCs have been made using SCNT; human ESCs have potential applications in both medicine and research.

Evans and a colleague discovered embryonic stem cells (often referred to as ES cells) in mice. These stem cells are derived from the inner cell mass of a mammalian embryo at a very early stage of development. After determining that ES cells could serve as vehicles for the transmission of

in the late 1980s with embryonic stem cells. Wilmut and his colleagues were interested primarily in nuclear transfer, a technique first conceived in 1928 by German embryologist Hans Spemann. Nuclear transfer involves the introduction of the nucleus from a cell into an enucleated egg cell (an egg cell that has

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Embryonic Stem Cell | California’s Stem Cell Agency

Diabetes mellitus currently afflicts approximately 370 million people worldwide, with projections of over 550 million by the year 2030 (sources: World Health Organization; International Diabetes Federation). In the year 2000 there were approximately 2 million cases of diabetes in California (source: Diabetes Control Program, California Department of Health Services). Further, the disease disproportionately affects certain minority groups and the elderly. Despite the use of insulin and advances in its delivery, the human cost of diabetes is underscored by the financial costs to society: tens of billions of dollars each year in California alone. The primary cause of type 1 diabetes, and contributing significantly to type 2 diabetes as well, is the loss of insulin-producing pancreatic beta cells. The CIRM Diabetes Disease Team Project is developing an innovative beta cell replacement therapy for insulin-requiring diabetes. If successful, the therapy will go beyond insulin function, and will perform the full array of normal beta cell functions, including responding in a more physiological manner than manual or mechanized insulin self-administration. Because they will be more physiological, the replacement cells could reduce the long-term effects of diabetes. Moreover, the cell therapy will alleviate patients of the constant monitoring of blood glucose, painful insulin injections, and the ever-present risk of overdosing with insulin. For these reasons, it is possible that the product could transform the diabetes treatment landscape dramatically and even replace pharmaceutical insulin in the market. This product will be available in California first, through clinical testing, and if approved by the FDA for commercial production, will eventually help hundreds of thousands of Californians with diabetes. The product will substantially increase quality of life for patients and their families, while significantly reducing the health care burden in the state. The proposed project will employ Californian doctors and scientists, and success will prove highly noteworthy for the state. Lastly, once commercially marketed, the product will yield additional jobs in manufacturing, sales, and related industries, and generate revenue for California. Given the market need and the clear feasibility, the product could become the most significant stem cell-based medical treatment of the coming decade, and that will be a tremendous achievement for California, its taxpayers, and CIRM.

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Making blood on demand: How far have we come? – Science Codex

The reconstitution of the blood system in humans holds great therapeutic potential to treat many disorders, like blood cancers, sickle-cell anemia and others. Successful reconstitution requires the transplantation and engraftment of hematopoietic (or blood) stem cells (HSCs), which after reaching their niche, start producing all types of blood cells, including platelets, white and red blood cells.

In current clinical practice, this is carried out by infusing HSCs obtained from a matched donor who is immunologically compatible with the patient in need (allogeneic transplantation), or by the expansion of the patient's own HSCs in the lab, and then re-infusing them back into the patient (ex-vivo, autologous transplantation). However, the utility of both routes is currently limited by a number of factors. First, in the case of allogeneic transplantation, the scarcity of matched donors significantly increases the waiting time, which could be detrimental to the patient. Second, the ex vivo expansion of HSCs, whether allogeneic or autologous, has been a challenging task, due to the limited proliferative potential these cells exhibit in culture. These limitations have raised the need for other sources of HSCs that would alleviate the need for matched donors and yield functional HSCs in large quantities.

In 2007, Professor Shinya Yamanaka and colleagues demonstrated that somatic cells, like skin fibroblasts, could be reprogrammed back to a cellular state that resembled human embryonic stem cells (hESCs), which are a group of cells found in the blastocyst-stage human embryo and contribute solely to the development of the human fetus during pregnancy. The reprogrammed cells were termed, Induced Pluripotent Stem Cells (iPSCs). In addition to their developmental potential, human ESCs and iPS cells display unlimited proliferative potential in culture, which makes them an ideal source of cells for regenerative medicine in general and for hematopoietic differentiation to obtain possibly unlimited quantities of HSCs. Therefore, there has been a growing interest to harness the potential of these cells for treating blood disorders.

However, advancement in deriving functional HSCs from human pluripotent stem cells has been slow. This has been attributed to incomplete understanding of the molecular mechanisms underlying normal hematopoiesis. In this review, the authors discuss the latest efforts to generate HSCs capable of long-term engraftment and reconstitution of the blood system from human pluripotent stem cells. Stem cell research has witnessed milestone achievements in this area in the last couple of years, the significance of which are discussed and analyzed in detail.

The authors additionally discuss two highly important families of transcription factors in the context of hematopoiesis and hematopoietic differentiation, the Homeobox (HOX) and GATA proteins. These are thought of as master regulators, in the sense of having numerous transcriptional targets, which upon activation, could elicit significant changes in cell identity. The authors hypothesize that precise temporal control of the levels of certain members of these families during hematopoietic differentiation could yield functional HSCs capable of long-term engraftment.

The authors conclude the review with a summary of future perspectives, in which they discuss how newly developed techniques, like the deactivated-Cas9 (dCas9) gene-expression control system, can be utilized during the course of hematopoietic differentiation of pluripotent stem cells for precise temporal control of the aforementioned master regulators to achieve functional HSCs.

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Making blood on demand: How far have we come? - Science Codex

Stem Cells Market Key Opportunities and Forecast up to 2025 – AnalyticSP

In theglobalstem cells marketa sizeable proportion of companies are trying to garner investments from organizations based overseas. This is one of the strategies leveraged by them to grow their market share. Further, they are also forging partnerships with pharmaceutical organizations to up revenues.

In addition, companies in the global stem cells market are pouring money into expansion through multidisciplinary and multi-sector collaboration for large scale production of high quality pluripotent and differentiated cells. The market, at present, is characterized by a diverse product portfolio, which is expected to up competition, and eventually growth in the market.

Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

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As per a report by Transparency Market Research, the global market for stem cells is expected to register a healthy CAGR of 13.8% during the period from 2017 to 2025 to become worth US$270.5 bn by 2025.

Depending upon the type of products, the global stem cell market can be divided into adult stem cells, human embryonic stem cells, induced pluripotent stem cells, etc. Of them, the segment of adult stem cells accounts for a leading share in the market. This is because of their ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Depending upon geography, the key segments of the global stem cells market are North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. At present, North America dominates the market because of the substantial investments in the field, impressive economic growth, rising instances of target chronic diseases, and technological progress. As per the TMR report, the market in North America will likely retain its dominant share in the near future to become worth US$167.33 bn by 2025.

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Investments in Research Drives Market

Constant thrust on research to broaden the utility scope of associated products is at the forefront of driving growth in the global stem cells market. Such research projects have generated various possibilities of different clinical applications of these cells, to usher in new treatments for diseases.Since cellular therapies are considered the next major step in transforming healthcare, companies are expanding their cellular therapy portfolio to include a range of ailments such as Parkinsons disease, type 1 diabetes, spinal cord injury, Alzheimers disease, etc.

The growing prevalence of chronic diseases and increasing investments of pharmaceutical and biopharmaceutical companies in stem cell research are the key driving factors for the stem cells therapeutics market. The growing number of stem cell donors, improved stem cell banking facilities, and increasing research and development are other crucial factors serving to propel the market, explains the lead analyst of the report.

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Stem Cells Market Key Opportunities and Forecast up to 2025 - AnalyticSP

My agonising two-year wait for a stem-cell donor after being diagnosed with leukaemia – The Telegraph

There is also another option on the table: a technology called haplo-identical, where they could use the stem cells from my brother, who is a 50 per cent match.

But it shouldnt have been this hard to find a match, and thats whyI started my campaign to sign more people up to the transplant list.I want to make a difference for other people who have to go through this.

If I dont make it, I want to leave a legacy that the children can look at when theyre older and know that Mummy did everything she could to fight this thing. There can only be one winner with this disease, and it needs to be me.

As told to Jessica Salter

Leukaemia Care is one of three charities supported by this years Telegraph Christmas Charity Appeal. Our others are Wooden Spoon, which works with the rugby community to raise money for disabled and disadvantaged children,and The Silver Line, a telephone support service for lonely elderly people. To donate,visit telegraph.co.uk/charity or call 0151 284 1927 before the end of January

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Top 10 Parkinson’s Stories of 2019 – Parkinson’s News Today

Parkinsons News Today keeps you up-to-date with research into Parkinsons disease as it emerges. We brought you daily coverage of experiments into the basic biology of Parkinsons, results of clinical and pre-clinical trials, and key findings from Parkinsons research around the globe.

We look forward to bringing more such news to those with Parkinsons, their family, friends and caregivers throughout 2020.

Here are our 10 most-read stories of 2019, with a short summary of what makes each one relevant to the Parkinsons community.

No. 10 Active Form of Vitamin B12 Found to Prevent Neurodegeneration in Study of Animal Models

A study found that an active form of the vitamin B12 called AdoCbl can ease the effects of dopamine loss that occurs in Parkinsons disease. Using cell lines and several animal models, researchers showed that by reducing LRRK2 enzyme activity, AdoCbl limits the death of dopamine-producing nerve cells, thereby preventing the appearance of symptoms associated with neurodegeneration. Overactivity of LRRK2 is linked to the development of a hereditary form of Parkinsons. AdoCbl is already an FDA-approved compound, and could be used as a basis to develop new therapies to combat hereditary Parkinsons associated with pathogenic variants of the LRRK2 enzyme, according to Iban Ubarretxena, director of theBiofisika Institute and a study co-author.

No. 9 Onstryv Now Approved for Parkinsons Patients in Canada

Canadian Parkinsons disease patients now have access to Onstryv (safinamide), also known as Xadago. Onstryv increases the amount of active dopamine in the brain by both preventing the enzyme that breaks dopamine down from doing so, and by blocking that enzyme from entering cells. Other available treatments cause debilitating fluctuations between normal motor function (called on episodes) and poorer motor function (off periods) as their effects ebb and flow. Four placebo-controlled Phase 3 trials showed that the combination of Onstryv and levodopa led to more on and fewer off periods, and improved motor function in patients. The approval of [Onstryv] in Canada is a step forward for patients who need new treatment options for Parkinsons disease, said Roberto Tascione, CEO of Zambon, one of the companies involved in commercializing this medication.

Xadago was approvedby theU.S. Food and Drug Administration in March 2017 to improvemotor functionin Parkinsons patients who experience off periods while on treatment withlevodopaand/or Lodosyn (carbidopa).

No. 8 Plant Antioxidant Seen to Aid Mitochondria and Ease Motor Problems in Early Parkinsons Study

A pre-clinical study conducted in China showed that alpha-arbutin, an antioxidant found in plants such as the blueberry, might restore mitochondrial function in nerve cells and ease the motor disabilities associated with Parkinsons disease. Treatment with alpha-arbutin partially restored mitochondrial function in nerve cells undergoing oxidative stress (mitochondria act as a cells power house). It also restored these cells ability to remove toxic waste products. Feeding alpha-arbutin to flies carrying a mutated gene known to trigger Parkinsons significantly eased several Parkinsons-like symptoms. Naturally derived-antioxidants might serve as a new class of therapeutic options for [Parkinsons disease], the researchers wrote.

No. 7 Stem Cell Transplants Could Significantly Improve Parkinsons Treatment, Study Suggests

Replacing damaged cells in Parkinsons disease with dopamine-producing stem cells could easemotor symptoms and reduce or eliminate the need for pharmaceutical medicines. As current disease therapies lose their efficacy over time, stem cell therapy might revolutionize Parkinsons treatment, its researchers said.A single surgery could potentially provide a transplant that would last throughout a patients lifespan, reducing or altogether avoiding the need for dopamine-based medications, said Claire Henchcliffe MD, PhD, and Malin Parmar, PhD, co-authors of a study on the benefits of stem cell therapy. However, there are several biological, practical, and commercial hurdles that need circumventing for this to become a routine therapy, according to the editors of theJournal ofParkinsons Disease.

No. 6 Bacteria in Gut Can Promote Parkinsons by Altering Brains Immune Reactions, Study Says

A study found evidence of interaction between the brain and the gut in Parkinsons, in which Gram-negative bacterial infections in the gut trigger an immune response that damages nerve cells. Gut microorganisms are known to communicate with the central nervous system, andstudies suggest that harmful proteins related to Parkinsons may spread to the brain from the gut.Scientists at theUniversit de Montral showed that Gram-negative bacteria, particularly those related to gut infections, triggered an immune response in cells taken from mice. They then showed that mice bred without thePINK1 gene (making them resistant to Parkinsons-like symptoms), when infected with these bacteria, displayed an immune response that led to such symptoms. Mutations in the PINK1 gene cause damage to the mitochondria in brain cells, and are linked to early onsethereditary Parkinsons. The work provides evidence that intestinal infection acts as a triggering event in Parkinsons, andhighlighted the relevance of a gut-brain connection in this disease.

No. 5 Next 20 Years Expected to Bring Message of Hope to Parkinsons Patients, Review Study Finds

By reviewing the past 20 years of research into Parkinsons disease, two scientists see a strong potential for breakthroughs in how this disease is approached over the next 20 years. The review cited developments in better animal models, greater understanding of molecular mechanisms and risk factors, and advances in available and potential therapies as reasons for hope. Among highlights of many advances listed are: 1) the adaptation of existing medicines used in other diseases to treat Parkinsons (drug repurposing); 2) targeting non-motor features such as cognition, speech and balance difficulties that often precede motor symptoms; 3) the use of nanoparticles to block the formation of toxic alpha-synuclein clusters; and, 4) emerging evidence of a link between harmful gut bacteria and brain inflammation. The review also stressed the importance of future trials to test combination therapies.

No. 4 Physical Activity, Coffee, Moderate Alcohol Consumption Protect Against Disease Progression, Study Reports

Good news for lovers of sports, caffeine, and happy hours all of these things, in moderation, may help slow the onset of symptoms of Parkinsons disease. Although how exactly these lifestyle factors affect disease progression remains poorly understood, they correlate strongly with better patient outcomes. Conversely, smoking, heavy drinking and no consumption of alcohol at all were linked to considerably worse outcomes. The study, published in the journalMovement Disorders, needs to be replicated to strengthen the usefulness of its findings. Nonetheless, the work suggests that multiple lifestyle factors potentially modify the rate of symptom progression, its researchers wrote.

No. 3 Dietary Supplement Eases Parkinsons Symptoms, Improves Dopamine Function, Study Shows

The antioxidant dietary supplement N-acetyl-cysteine (NAC) may improve dopamine function and ease Parkinsons disease symptoms, according to one study. The body uses NAC to produce an antioxidant called glutathione (GSH), which it uses to prevent the oxidative stress that leads to cell death. Damage due to oxidative stress within dopamine-producing neurons is a key clinical feature of Parkinsons. A trial (NCT02445651), conducted by researchers atThomas Jefferson University in Philadelphia, showed that NAC supplementation significantly eased both motor andnon-motor symptoms among 42 Parkinsons patients (21 men and 21 women). These results need to be confirmed in larger and placebo-controlled studies, but offer an encouraging start to a potential low-cost therapy.

No. 2 Low Vitamin D Levels Linked to Added Falls, More Sleep Problems, Depression, Study Shows

Low levels of vitamin D were associated with more falls, and greater problems withinsomnia, anxiety, anddepressionin people withParkinsons disease, according to a study by Chinese researchers. Vitamin D deficiency has often been seen in people with Parkinsons, but its relationship to the disease remains controversial. This study, by researchers at theSecond Affiliated Hospital of Soochow UniversityandSoochow University, is one of the few to measure both motor and non-motor outcomes. By conducting detailed clinical evaluations in 182 Parkinsons patients, as well as 185 healthy controls, the group found that low levels of vitamin D were more common in Parkinsons patients than in healthy people, and that vitamin D supplements may ease the diseases nonmotor symptoms.

No. 1 Oral Magnesium Compound Able to Reach Brain Seen to Slow Motor Decline, Neuronal Loss in Early Study

Our years most-read story was of an early stage studyreporting that a type of oral magnesium could enter the brain and ease motor symptoms and nerve cell loss in a mouse model of Parkinsons disease. Mice given magnesium-L-threonate, which can cross the blood-brain barrier (a semipermeable membrane that protects the brain from the outside environment) reduced the loss of dopamine-producing neurons, slowed the decline in motor function, and limited the oxidative stress that is associated with Parkinsons. It is important to note that while magnesium-L-threonate provided therapeutic benefits, magnesium sulfate the first choice as a clinical magnesium supplement did not. [T]he combination of [magnesium] with an agent that promotes its transportation to the brain is essential for the neuroprotection of this element, the studys scientists wrote.

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AtParkinsons News Today we hope these stories and our reporting throughout 2020 help to better inform and improve the lives of everyone affected by Parkinsons.

We wish all our readers a happy 2020.

Total Posts: 208

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Top 10 Parkinson's Stories of 2019 - Parkinson's News Today