Category Archives: Induced Pluripotent Stem Cells


Is it time to start worrying about conscious human mini-brains? – PLoS Blogs (blog)

A human iPSC cerebral organoid in which pigmented retinal epithelial cells can be seen (from the work of McClure-Begley, Mike Klymkowsky, and William Old.)

The fact that experiments on people are severely constrained is a major obstacle in understanding human development and disease. Some of these constraints are moral and ethical and clearly appropriate and necessary given the depressing history of medical atrocities. Others are technical, associated with the slow pace of human development. The combination of moral and technical factors has driven experimental biologists to explore the behavior of a wide range of model systems from bacteria, yeasts, fruit flies, and worms to fish, frogs, birds, rodents, and primates. Justified by the deep evolutionary continuity between these organisms (after all, all organisms appear to be descended from a single common ancestor and share many molecular features), experimental evolution-based studies of model systems have led to many therapeutically valuable insights in humans something that I suspect a devotee of intelligent design creationism would be hard pressed to predict or explain (post link).

While humans are closely related to other mammals, it is immediately obvious that there are important differences after all people are instantly recognizable from members of other closely related species and certainly look and behave differently from mice. For example, the surface layer of our brains are extensively folded (they are known as gyrencephalic) while the brain of a mouse is smooth as a babys bottom (and referred to as lissencephalic). In humans, the failure of the brain cortex to fold is known as lissencephaly, a disorder associated with several severe neurological defects. With the advent of more and more genomic sequence data, we can identify human specific molecular (genomic) differences. Many of these sequence differences occur in regions of our DNA that regulate when and where specific genes are expressed. Sholtis & Noonan (1) provide an example: the HACNS1 locus is a 81 basepair region that is highly conserved in various vertebrates from birds to chimpanzees; there are 13 human specific changes in this sequence that appear to alter its activity, leading to human-specific changes in the expression of nearby genes (). At this point ~1000 genetic elements that are different in humans compared to other vertebrates have been identified and more are likely to emerge (2). Such human-specific changes can make modeling human-specific behaviors, at the cellular, tissue, organ, and organism level, in non-human model systems difficult and problematic (3,4). It is for this reason that scientists have attempted to generate better human specific systems.

One particularly promising approach is based on what are known as embryonic stem cells (ESCs) or pluripotent stem cells (PSCs). Human embryonic stem cells are generated from the inner cell mass of a human embryo and so involve the destruction of that embryo which raises a number of ethical and religious concerns as to when life begins (5)(more on that in a future post). Human pluripotent stem cells are isolated from adult tissues but in most cases require invasive harvesting methods that limit their usefulness. Both ESCs and PSCs can be grown in the laboratory and can be induced to differentiate into what are known as gastruloids. Such gastruloids can develop anterior-posterior (head-tail), dorsal-ventral (back-belly), and left-right axes analogous to those found in embryos (6) and adults (top panel). In the case of PSCs, the gastruloid (bottom panel ) is essentially a twin of the organism from which the PSCs were derived, a situation that raises difficult questions: is it a distinct individual, is it the property of the donor or the creation of a technician. The situation will be further complicated if (or rather, when) it becomes possible to generate viable embryos from such gastruloids.

The Nobel prize winning work of Kazutoshi Takahashi and Shinya Yamanaka (7), who devised methods to take differentiated (somatic) human cells and reprogram them into ESC/PSC-like cells, cells known as induced pluripotent stem cells (iPSCs)(8), represented a technical breakthrough that jump-started this field. While the original methods derived sample cells from tissue biopsies, it is possible to reprogramkidney epithelial cells recovered from urine, a non-invasive approach (9,10). Subsequently, Madeline Lancaster, Jurgen Knblich, and colleagues devised an approach by which such cells could be induced to form what they termed cerebral organoids; they used thismethod to examine the developmental defects associated with microencephaly (11). The value of the approach was rapidly recognized and a number of studies on human conditions, including lissencephaly (12), Zika-virus infection-induced microencephaly(13), and Downs syndrome (14); investigatorshave begun to exploit these methodsto study a range of human diseases.

The production of cerebral organoids from reprogrammed human somatic cells has also attracted the attention of the media (15). While mini-brain is certainly a catchier name, it is a less accurate description of a cerebral organoid, itself possibly a bit of an overstatement, since it is not clear exactly how cerebral such organoids are. For example, the developing brain is patterned by embryonic signals that establish its asymmetries; it forms at the anterior end of the neural tube (the nascent central nervous system and spinal cord) and with distinctive anterior-posterior, dorsal-ventral, and left-right asymmetries, something that simple cerebral organoids do not display. Moreover, current methods for generating cerebral organoids involve primarily what are known as neuroectodermal cells our nervous system (and that of other vertebrates) is a specialized form of the embryos surface layer that gets internalized during development. In the embryo, the developing neuroectoderm interacts with cells of the circulatory system (capillaries, veins, and arteries), formed by endothelial cells and what are known as pericytes that surround them. These cells, together with interactions with glial cells (astrocytes, a non-neuronal cell type) combine to form the blood brain barrier. Other glial cells (oligodendrocytes) are also present; in contrast, both types of glia (astrocytes and oligodendrocytes) are rare in the current generation of cerebral organoids. Finally, there are microglial cells, immune system cells that originate from outside the neuroectoderm; they invade and interact with neurons and glia as part of the brains dynamic neural system. The left panel of the figure shows, in highly schematic form how these cells interact (16). The right panel is a drawing of neural tissue stained by the Golgi method (17), which reveals~3-5% of the neurons present. There are at least as many glial cells present, as well as microglia, none of which are visible in the image. At this point, cerebral organoids typically contain few astrocytes and oligodendrocytes, no vasculature, and no microglia. Moreover, they grow to be about 1 to 3 mm in diameter over the course of 6 to 9 months; that is significantly smaller in volume than a fetal or newborns brain. While cerebral organoids can generate structures characteristic of retinal pigment epithelia (top figure) and photo-responsive neurons (18), such as those associated with the retina, an extension of the brain, it is not at all clear that there is any significant sensory input into the neuronal networks that are formed within a cerebral organoid, or any significant outputs, at least compared to the role that the human brain plays in controlling bodily and mental functions.

The reasonable question, then, must be whether a cerebral organoid, which is a relatively simple system of cells (although itself complex), is conscious. It becomes more reasonable as increasingly complex systems are developed, and such work is proceeding apace. Already researchers are manipulating the developing organoids environment to facilitate axis formation, and one can anticipate the introduction of vasculature. Indeed, the generation of microglia-like cells from iPSCs has been reported; such cells can be incorporated into cerebral organoids where they appear to respond to neuronal damage in much the same way as microglia behave in intact neural tissue (19).

We can ask ourselves, what would convince us that a cerebral organoid, living within a laboratory incubator, was conscious? How would such consciousnessmanifest itself? Through some specific pattern of neural activity, perhaps? As a biologist, albeit one primarily interested in molecular and cellular systems, I discount the idea, proposed by some physicists and philosophers as well as the more mystical, that consciousness is a universal property of matter (20,21). I take consciousness to be an emergent property of complex neural systems, generated by evolutionary mechanisms, builtduring embryonic and subsequent development, and influenced bysocial interactions (BLOG LINK) using information encoded within the human genome (something similar to this: A New Theory Explains How Consciousness Evolved). While a future concern, in a world full of more immediate and pressing issues, it will be interesting to listen to the academic, social, and political debate on what to do with mini-brains as they grow in complexity and perhaps inevitably, towards consciousness.

Footnotes and references

Thanks to Rebecca Klymkowsky, Esq. and Joshua Sanes, Ph.D. for editing anddisciplinarysupport.

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Is it time to start worrying about conscious human mini-brains? - PLoS Blogs (blog)

SBP Scientist Receives Prestigious WM Keck Foundation Grant – Newswise (press release)

Newswise La Jolla, Calif., July 10, 2017 Sanford Burnham Prebys Medical Discovery Institute (SBP) is pleased to announce that the W.M. Keck Foundation has awarded a $1M grant to Duc Dong, Ph.D., to advance research to generate replacement organs and cellssuch as insulin producing cells for diabetes or dopamine-producing neurons for Parkinsons diseasedirectly in the body and without the use of stem cells.

The grant will allow Dong to extend his studies reprogramming dispensable cells such as skin, vasculature and fat cells while they remain in the body, in vivo, without the use of cultures or induced pluripotent stem cell techniques, which could be a safer and more effective method to treat degenerative diseases and injuries.

Dong, an assistant professor in the Human Genetics Program, uses zebrafishtiny striped fish commonly used for genetic studies-as a vertebrate model to reprogram cells into unrelated types, completely within the body of these living animals. Dongs research team already has proof-of-concept data demonstrating in vivo conversion of muscle and skin cells into beta-cell precursors (the cells that mature to produce insulin)an accomplishment assumed to be impossible by most scientists.

Im honored to have received this grant from the Foundation, says Dong. The award will support my vision to ultimately make in vivo cell lineage reprogramming a practical therapeutic strategy to replace or even enhance lost, damaged or aging tissues. Our next steps are to develop technologies to convert any cells in the body, at any age, into any cell type of interest, and to uncover the molecular mechanisms driving that process.

Based in Los Angeles, the W. M. Keck Foundation was established in 1954 by the late W. M. Keck, founder of the Superior Oil Company. The Foundations grant making is focused primarily on pioneering efforts in the areas of medical research, science and engineering and undergraduate education. The Foundation also maintains a Southern California Grant Program thatprovides support for the Los Angeles community, with a special emphasis on children and youth. For more information, please visit http://www.wmkeck.org.

About SBPSanford Burnham Prebys Medical Discovery Institute (SBP) is an independent nonprofit medical research organization that conducts world-class, collaborative, biological research and translates its discoveries for the benefit of patients. SBP focuses its research on cancer, immunity, neurodegeneration, metabolic disorders and rare childrens diseases. The Institute invests in talent, technology and partnerships to accelerate the translation of laboratory discoveries that will have the greatest impact on patients. Recognized for its world-class NCI-designated Cancer Center and the Conrad Prebys Center for Chemical Genomics, SBP employs about 1,100 scientists and staff in San Diego (La Jolla), Calif., and Orlando (Lake Nona), Fla. For more information, visit us at SBPdiscovery.org or on Facebook at facebook.com/SBPdiscovery and on Twitter @SBPdiscovery.

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SBP Scientist Receives Prestigious WM Keck Foundation Grant - Newswise (press release)

Grnenthal Group: Launch of the Project – Modelling Neuron-glia Networks Into a Drug Discovery Platform for Pain … – PR Newswire (press release)

The new research project NGN-PET was launched in the framework of the Innovative Medicine Initiative (IMI), the largest public-private partnership (PPP) for health research worldwide co-funded by the EU and the European pharmaceutical industry. The NGN-PET consortium unites the expertise and knowledge of industry partners from the European Federation of Pharmaceutical Industries and Associations (EFPIA), small and medium-sized enterprises (SMEs) and academia. Together they will investigate neuron-glia interactions aiming to develop authentic cellular (co-culture) assays to discover improved treatments of neuropathic pain using neuronal and glial cell types derived from human induced pluripotent stem cells (iPSC), and their co-cultures.

The primary objective is to provide a translational platform for the identification, validation and testing of neuropathic pain targets in preclinical and human-relevant test systems suitable for drug discovery.

Chronic pain is a serious debilitating disease which greatly reduces the quality of life for the individual patients. In Europe, 20% of the population are affected which causes considerable socioeconomic burden of over 200 bn per year[1]. Chronic pain of neuropathic origin has a population prevalence of 8.2%[2].

Neuropathic pain arises after insults such as surgery, trauma, diabetes, chemotherapy or viral infections, and its prevalence is expected to rise in the future due to the ageing society.

Current treatments for chronic pain have limited efficacy, leaving about 60% of patients without adequate pain relief[1]. Moreover, these therapies address only symptoms not the causes of the pain, and are therefore not curative. In fact, the aetiologies of the disease are poorly understood which hinders the development of new analgesics with improved efficacies.

One of the major findings of the last decade in pain research is that non-neuronal cells play a very active role in the development of sensory abnormalities. In particular, glia - like Schwann cells, microglia, or astrocytes - contribute directly to modulation of neuronal functions.

NGN-PET consortium: understand biology and develop test systems for neuropathic pain

The NGN-PET consortium aims to explore neuron-glial interactions in subtypes of neuropathic pain which are induced by chemotherapy or trauma, and to develop human-predictive test systems that can be implemented in the drug discovery process. These cellular systems will use preclinical tissues and human iPSC-derived neuron-glia co-cultures in novel high-throughput screening platforms. We hope this new science helps in identifying novel more efficacious treatments for neuropathic pain patients.

To achieve these ambitious goals, a consortium of 6 partners, with the support of IMI, has been formed. NGN-PET brings together experts from industry, SMEs and academia in a synergistic public-private partnership. NGN-PET is supported by over 3million euros from IMI2 and industry partners in direct and in-kind contributions. The project duration is 3 years. The consortium will disseminate the results through publication in high-impact scientific journals, applying open access policy whenever possible, or in scientific meetings by means of poster or oral presentations. Online outreach of the project publications will be performed via the project website.

The NGN-PET project is coordinated by Axxam; the project leader is ESTEVE, supported by Grnenthal as project Co-Leader.

About the partners

About Axxam

Axxam is an innovative Partner Research Organization (iPRO) based in Milan, Italy. Axxam is a leading provider of integrated discovery services across Life Sciences industries including: pharmaceuticals, crop protection, animal health, cosmetics, fragrances, food and beverages. The company has consolidated expertise across a broad range of discovery disciplines and innovative technologies including: assay development, high-throughput screening of both the Axxam high quality compound collections (synthetic and natural) or those provided by our clients, compound management, hit identification and hit validation. Axxam performance-driven approach has been recognized by the clients as key to the success for their discovery programs. Axxam is also engaged in alliance-based research towards innovative small molecule therapies for diseases with a high unmet medical need. Axxam's business terms are flexible, ranging from fee-for-service to risk-sharing deal structures.

More information: http://www.axxam.com

About Life & Brain

LIFE & BRAIN GmbH is a biomedical enterprise founded in 2002 and located at the University Hospital Campus in Bonn, Germany. As a center of innovation, LIFE & BRAIN acts as a revolving door between academic research and industry. Innovative research results are recognized early and developed further into marketable biomedical products and services. Its mission is to discover and develop novel strategies for the diagnosis and therapy of nervous system disorders. A key focus of LIFE & BRAIN is the development and provision of human pluripotent stem cell-based tools and services for neurological disease modeling and drug discovery. Within the project LIFE & BRAIN will provide induced pluripotent stem cell-derived glial and neuronal populations to model the neuron-glia network in neuropathological pain conditions.

More information: http://www.lifeandbrain.com

About King's College London

King's College London is one of the top 25 universities in the world (2016/17 QS World University Rankings) and among the oldest in England. Research at King's has played a major role in many of the advances that have shaped modern life, such as the discovery of the structure of DNA and work that led to the development of radio, television, mobile phones and radar.

King's has more than 26,500 students from some 150 countries world-wide and nearly 6,900 staff. The university has an outstanding reputation for world-class teaching and cutting-edge research. King's was ranked 6th nationally in the 2014 Research Excellence Framework (REF) and is in the top seven UK universities for research earnings with an overall annual income of more than 600 million.

More information: http://www.kcl.ac.uk

About NMI

The Natural and Medical Sciences Institute at the University of Tbingen (NMI) is a member of the Innovation Alliance Baden-Wrttemberg. Its main activities focus on application-oriented research at the interface between life and material sciences. In addition, it also operates as business incubator for start-up companies. NMI unique and interdisciplinary spectrum of skills and competencies, supported by a strong team of more than 150 scientists, provides an ideal research environment where innovative technologies are brought together for the benefit of public stakeholders and industry. A broad range of thematic areas are covered across several departments and laboratories:

- Pharma & biotechnology: targets and biomarkers for the identification of active compounds, electrophysiology, diagnostics and bio-analytics

- Biomedical engineering: implants, biosensors, biomaterials and regenerative medicine

- Surface and material technology: micro and nano-analytics, coatings, adhesive bonding systems

More information: http://www.nmi.de

About Esteve

Esteve is a leading pharmaceutical chemical group based in Barcelona, Spain. Since it was founded in 1929, Esteve has been firmly committed to excellence in healthcare, dedicating efforts to innovative R&D of new medicines for unmet medical needs and focusing on high science and evidencebased research. Esteve has a strong partnership approach to drug discovery, development and commercialisation. The company works both independently and in collaboration to bring new, differentiated bestinclass treatments to patients. The company currently employs 2,300 professionals and has subsidiaries and production facilities in several European countries, USA, China and Mexico.

More information: http://www.esteve.es

About Grnenthal

The Grnenthal Group is an entrepreneurial, science-based pharmaceutical company specialized in pain, gout and inflammation. Our ambition is to deliver four to five new products to patients in diseases with high unmet medical need by 2022 and become a 2 billion company. We are a fully integrated research & development company with a long track record of bringing innovative pain treatments and state-of-the-art technologies to patients. By sustainably investing in our R&D above the industrial average, we are strongly committed to innovation.

Grnenthal is an independent, family-owned company headquartered in Aachen, Germany. We are present in 32 countries with affiliates in Europe, Latin America and the US. Our products are sold in more than 155 countries and approx. 5,500 employees are working for the Grnenthal Group worldwide. In 2016, Grnenthal achieved revenues of approx. 1.4 bn.

More information: http://www.grunenthal.com

About the Innovative Medicines Initiative

The Innovative Medicines Initiative (IMI) is working to improve health by speeding up the development of, and patient access to, the next generation of medicines, particularly in areas where there is an unmet medical or social need. It does this by facilitating collaboration between the key players involved in healthcare research, including universities, pharmaceutical companies, and other companies active in healthcare research, small and medium-sized enterprises (SMEs), patient organisations, and medicines regulators. This approach has proven highly successful, and IMI projects are delivering exciting results that are helping to advance the development of urgently-needed new treatments in diverse areas.

IMI is a partnership between the European Union and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA). Through the IMI 2 programme, IMI has a budget of 3.3 bn for the period 2014-2024. Half of this comes from the EU's research and innovation programme, Horizon 2020. The other half comes from large companies, mostly from the pharmaceutical sector; these do not receive any EU funding, but contribute to the projects 'in kind', for example by donating their researchers' time or providing access to research facilities or resources.

Contact Project Office/General Enquires Email: info@ngn-pet.com Project Leader: Xavier Codony , ESTEVE. +34-93-4466061 Project Coordinator: Dr. Paola Tarroni, Axxam SpA. +39-02-2105639 Project Manager: Dr. Enric Castells, ESTEVE. +34-93-4466112

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 116072. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA Companies.

[1] van Hecke O, Torrance N, Smith BH. Chronic pain epidemiology and its clinical relevance. Br J Anaesth. 2013; 111(1):13-8. [2] Torrance N, Smith BH, Bennett MI, Lee AJ. The epidemiology of chronic pain of predominantly neuropathic origin. Results from a general population survey. J Pain. 2006 Apr;7(4):281-9.

SOURCE Grnenthal Group

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Grnenthal Group: Launch of the Project - Modelling Neuron-glia Networks Into a Drug Discovery Platform for Pain ... - PR Newswire (press release)

The Global Market for Induced Pluripotent Stem Cells (iPSCs) should reach $3.6 Billion in 2021, Increasing at a CAGR … – Business Wire (press…

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Induced Pluripotent Stem Cells: Global Markets" report to their offering.

The Global Market for Induced Pluripotent Stem Cells (iPSCs) should reach $3.6 Billion in 2021, Increasing at a CAGR of 11.6% from 2016 through 2021

This study is focused on the market side of iPSCs rather than its technical side. Different market segments for this emerging market are covered.

For example, application-based market segments include academic research, drug development and toxicity testing, and regenerative medicine; product function-based market segments include molecular and cellular engineering, cellular reprogramming, cell culture, cell differentiation and cell analysis; iPSC-derived cell-type-based market segments include cardiomyocytes, hepatocytes, neurons, endothelia cells and other cell types; geography-based market segments include the U.S., Europe, Asia-Pacific and Rest of World. Research and market trends are also analyzed by studying the funding, patent publications and research publications in the field.

Key Topics Covered:

1: Introduction

2: Summary and Highlights

3: Overview

4: Induced Pluripotent Stem Cell Applications

5: Induced Pluripotent Stem Cell Market Segmentation and Forecast

6: Induced Pluripotent Stem Cell Research Application Market

7: Drug Discovery and Development Market

8: Induced Pluripotent Stem Cell Contract Service Market

9: Induced Pluripotent Stem Cell Clinical Application Market

10: Research Market Trend Analysis

11: Clinical Application Market Trend Analysis

12: Company Profiles

13: Conclusions

For more information about this report visit https://www.researchandmarkets.com/research/qwdtwm/induced

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The Global Market for Induced Pluripotent Stem Cells (iPSCs) should reach $3.6 Billion in 2021, Increasing at a CAGR ... - Business Wire (press...

This Study Could Help Extend the Human Lifespan – Futurism

In BriefResearchers have identified a single gene deletion in E. colibacteria that influence longevity in C. elegans worms. This pointsto the role of gut bacteria in life extension and points to thepossibility of a life-extending probiotic in the future.

Researchers at the Baylor College of Medicine have found the key to longevity in Caenorhabditis elegans (C. elegans) worms and maybe, someday, humans. The team noticed that genetically identical worms would occasionally live for much longer, and looked to their gut bacteria to find the answer. They discovered that a strain of E. coli with a single gene deletion might be the reason that its hosts lives were being significantly extended.

This study is one among a number of projects that focus on the influence of the microbiome the community of microbes which share the body of the host organism on longevity. Ultimately, the goal of this kind of research is to develop probiotics that could extend human life. Ive always studied the molecular genetics of aging, Meng Wang, one of the researchers who conducted the study, told The Atlantic. But before, we always looked at the host. This is my first attempt to understand the bacterias side.

Even in cases like this, where it seems fairly obvious that the microbiome is influencing longevity, parsing out the details of how and why this happens among a tremendous variety of chemicals and microbe species is extremely complex. The team, in this case, was successful because they simplified the question and focused on a single relationship.

Genetically engineering bacteria to support and improve human health and even to slow aging and turning it into a usable, life-extending probiotic wont be easy. It is extremely difficult to make bacteria colonize the gut in a stable manner, which is a primary challenge in this field. The team, in this case, is looking to the microbiome, because the organisms used would be relatively safe to use because they would originate in the gut.

Clearly, researchers dont know yet whether these discoveries will be able to be applied to people, though it seems promising. Despite the obvious differences between the tiny C. elegans worm and us, its biology is surprisingly similar; many treatments that work well in mice and primates also work in the worm. The team will begin experiments along these same lines with mice soon.

Other interesting and recent research hoping to stop or slow the march of time includes work with induced pluripotent stem (iPS) cells, antioxidants that target the mitochondria, and even somewhat strangework with cord blood. It seems very likely that we wont have a single solution offering immortality anytime soon, but instead a range of treatment options that help to incrementally hold back time. And, with an improving quality of life, this kind of life extension sounds promising.

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This Study Could Help Extend the Human Lifespan - Futurism

Embryonic stem cells to be available for medical use in Japan by next March – The Japan Times

KYOTO Embryonic stem cells for regenerative medicine will become available to medical institutions by the March 2018 end of fiscal 2017, Hirofumi Suemori, associate professor at Kyoto Universitys Institute for Frontier Life and Medical Sciences, has said.

Suemori also said Tuesday that ES cells for medical treatment, which have been approved by both the health and science ministries, would be created from October at the earliest.

To make ES cells, Kyoto University will work with Adachi Hospital in the city of Kyoto, which offers infertility treatment, to use fertilized eggs that would otherwise be disposed of.

The university hopes to obtain unwanted fertilized eggs from patients undergoing infertility treatment. The fertilized eggs are expected to be provided from around next February, the university said.

ES cells have the potential to become a variety of cell types much as induced pluripotent stem (iPS) cells do. Using ES cells, clinical trials are being conducted abroad on retinopathy, spinal cord injuries, Parkinsons disease, diabetes and cardiac disorders.

Many patients have qualms about discarding fertilized eggs, said Adachi Hospital Director Hiroshi Hatayama, who joined Suemori for a news conference. We can present an option to them, Hatayama said.

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Embryonic stem cells to be available for medical use in Japan by next March - The Japan Times

Treating Asthma with Stem Cells | Technology Networks – Technology Networks

A study led by scientists at Monash University has shown that a new therapy developed through stem cell technology holds promise as a treatment for chronic asthma.

The Monash Biomedicine Discovery Institute (BDI) scientists provided the experimental expertise to test Cynata Therapeutics induced pluripotent stem cell-derived mesenchymal stem cells (MSCs) in a model of experimental asthma. Induced pluripotent stem cells are a type of pluripotent stem cell that can be generated directly from adult cells; they have the ability to be differentiated into a variety of tissue types and, in this case, MSCs that can regenerate damaged lung tissue.

Lead researchers Associate Professor Chrishan Samuel and Dr Simon Royce tested the efficacy of the MSCs on three key components of asthma in a preclinical model of chronic allergic airways disease: inflammation; airway remodeling (structural changes that occur in lungs as a result of prolonged inflammation); and airway hyperresponsiveness (the clinical symptom of asthma).

The study, published in the FASEB Journal, found that the MSCs could effectively reduce inflammation, reversed signs of airway remodelling and completely normalised airway/lung fibrosis and airway hyperresponsiveness, particularly when delivered intranasally.

It concluded that they may provide a novel stand-alone therapy or an adjunct therapy for groups of asthma sufferers who do not respond to current (corticosteroid) therapy.

Most importantly, what we found was you can treat fibrosis (hardening or scarring of the lung) very effectively, said Associate Professor Samuel, who heads the Monash BDIs Fibrosis Laboratory.

When weve tested other types of stem cells they havent been able to fully reverse scarring and lung dysfunction associated with asthma weve had to combine them with anti-scarring drugs to achieve that. These cells were remarkable on their own as they were able to effectively reverse the scarring that contributes to lung dysfunction and difficulty in breathing, he said.

One in nine - or around 2.5 million- Australians have asthma.

Further research will be conducted to test the MSCs in combination with, or compared to a clinically-used corticosteroid. Clinical trials using the cells as a novel target for asthma are then envisaged.

Cynata Therapeutics Limited is an Australian clinical-stage stem cell and regenerative medicine company developing therapies based on its proprietary Cymerus stem cell technology platform.

This article has been republished frommaterialsprovided by Monash University. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Royce, S. G., Rele, S., Broughton, B. R., Kelly, K., & Samuel, C. S. (2017). Intranasal administration of mesenchymoangioblast-derived mesenchymal stem cells abrogates airway fibrosis and airway hyperresponsiveness associated with chronic allergic airways disease. The FASEB Journal. doi:10.1096/fj.201700178r

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Treating Asthma with Stem Cells | Technology Networks - Technology Networks

When C9ORF72 Silences U2, Spliceosomes Can’t Find What They … – Alzforum

30 Jun 2017

Expanded C9ORF72 hexanucleotide repeats cause many cases of amyotrophic lateral sclerosis and frontotemporal dementia, but exactly how these expansions work their mischief remains a mystery. Researchers have proposed several different pathogenic mechanisms, including that aberrant dipeptide repeat (DPR) proteins made from the expansion interfere with mRNA splicing. In the June 13 Cell Reports, researchers led by Robin Reed at Harvard Medical School describe how this could happen. Using in vitro assays, they found that DPRs prevented spliceosome assembly through interactions with one portion of this splicing complex, the U2 small nuclear ribonucleoprotein (snRNP) particle. In cells, C9ORF72 DPRs caused the U2 complex to mislocalize to the cytoplasm, away from splicing sites in the nucleus. Subsequent bioinformatics analyses suggested that disruption of U2 function accounts for about 40 percent of the mis-splicing seen in C9ORF72 patient cells, Reednoted.

The data for the mechanism are compelling, but it remains unclear how much it contributes to neurotoxicity, noted Johnathan Cooper-Knock at the University of Sheffield, England, in an email to Alzforum. He was not involved in the research. One way to address this might be to examine its correlation with clinical phenotype, he suggested (see full commentbelow).

In control motor neurons (left), the U2 splicing complex (red) occupies the nucleus, but in C9ORF72 motor neurons (right), much of it lurks in cytoplasm. [Courtesy of Cell Reports, Yin etal.]

Previous research had found that C9ORF72 expansions caused mis-splicing in cultured cells (see Aug 2014 news; Conlon et al., 2016). In one study, the degree of this mis-splicing correlated with faster ALS progression (Cooper-Knock et al., 2015).The papers proposed different explanations for how mis-splicing occurred, however, from dipeptide repeat proteins physically gumming up the splicing machinery in the nucleolus to C9ORF72-expanded RNAs sequestering crucial splicing factors such asHNRNPH.

To try to nail down the mechanism, first author Shanye Yin used a cell-free system developed in the Reed lab that combined nuclear extract from HeLa cells with a DNA template for the fly Ftz gene. In this assay, the Ftz gene is transcribed by RNA polymerase and the resulting RNA is then spliced to yield mature transcript. However, when the authors added a 20-repeat length of the toxic DPRs glycine-arginine (GR) or proline-arginine (PR), splicing ground to a halt. The effectiveness of the block depended on the dose of DPR. Further analysis showed that in the presence of DPRs, the spliceosome failed to assemble properly. C9ORF72 RNA, by contrast, had no effect on splicing in thissystem.

The authors next searched for proteins that associated with GR and PR using pull-down assays, and identified these interactors by mass spectrometry. The most common were components of the U2 snRNP, although other proteins were present as well. The list of interacting proteins largely matches that seen in previous proteomic screens of DPRs, noted Paul Taylor at St. Jude Childrens Research Hospital in Memphis, Tennessee. That gives us confidence in the data, he toldAlzforum.

The findings suggested a direct interaction of DPRs with the splicing machinery in vitro. Would the same thing happen in patients? The authors examined motor neurons made from induced pluripotent stem cells of people who carried the C9ORF72 expansion. They were surprised to find U2 snRNP lingering outside the nucleus in about half these cells (see image above). It was unclear if the complex leaked out of the nucleus, or simply never made it inside in the first place, Reed noted. U2 snRNP is assembled in the cytoplasm, and faulty transport across the nuclear membrane has quickly become a hallmark of C9ORF72 disease, with many nuclear proteins, such as TDP43 and FUS, getting stuck en route (Jan 2010 news;Aug 2015 news).DPRs are known to build up in the cytoplasm, suggesting they could be sequestering the U2 snRNP there. Overexpressing PR in HeLa cells also caused U2 snRNP to loiter in cytoplasm, supporting a direct causal role for these peptides in U2 mislocalization. This was specific to U2, as other components of the splicing apparatus stayed confined to thenucleus.

The authors next wondered how big a role U2 mis-splicing might play in disease. U2 snRNP normally helps splice about one-quarter of all transcripts (see Kfir et al., 2015). Analyzing published RNA expression data from C9ORF72 cerebella and frontal cortices (Prudencio et al., 2015), the authors found that the mis-spliced mRNA was enriched for U2-dependent transcripts. Altogether, U2 snRNP may account for nearly half of the mis-splicing in these cells, they calculated. They saw a much smaller role for HNRNPH-dependent splicing. Notably, in brain samples from sporadic ALS cases, U2-dependent transcripts were no more likely to be mis-spliced than any others, indicating this mechanism is specific for C9ORF72expansions.

In theory, mis-splicing could lead to toxicity by knocking out essential proteins. In support of this, the authors found numerous transcripts involved in mitochondrial function and gene expression among the disrupted set. Both of these processes are known to be perturbed inALS.

However, DPRs sequester other proteins as well, which may play an equally important role in damaging cell function, Taylor noted. Many of the proteins bound by DPRs contain low-complexity domains. Such proteins are now believed to be crucial in assembling many cellular organelles (Oct 2015 webinar; May 2017 conference news).Madolyn BowmanRogers

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When C9ORF72 Silences U2, Spliceosomes Can't Find What They ... - Alzforum

The Stem Cell Revolution – Seeking Alpha

By Manisha Samy, ARK analyst

Discovered nearly thirty years ago in 1988[1], stem cell technology has failed to transform the field of regenerative medicine until now. After delivering only one treatment during three decades of development, stem cell technology could finally become the all-purpose tool for repairing the body, thanks to rapid and precise genome editing techniques such as CRISPR and TALENs. Several obstacles in stem cell technology- high costs, safety concerns, and bioethical considerations are beginning to fall away. In particular, the introduction of induced pluripotent stem cells (iPSCs) eliminated many of the initial bioethical concerns stirred by the the source of stem cells. Now, advances in genome-editing is accelerating the pace of progress.

Stem cells exist in a state of possibility. Two key markers of stem cells are 1) the ability to self-renew and 2). The ability to become any specialized cell. Broadly speaking, a stem cell can evolve into any of the ~200 specialized human cell types, as illustrated in Figure 1. Directing stem cells to become any one of the 200 cell types has proven challenging.

Moreover, before the introduction of iPSCs, which are derived from adult cells, most human stem cells were sourced from human embryos or cord blood. The political and ethical controversies surrounding embryonic research curtailed stem cell funding. As a result, after a quarter century of research, bone marrow transplantation is the only FDA-approved stem cell application in the United States.

Figure 1

Breakthroughs in cheap, rapid genome-editing have re-invogored momentum in stem cell research. As shown in Figure 2, the number of publications on PubMed mentioning both stem cells and gene-editing hit a tipping point upon the discovery of CRISPR in 2012, three years after the first instance of precise TALENs-based genome-editing in human cells. Based on the current trajectory, ARK estimates that the number of scientific publications including both genome-editing and stem cells will approach 300 this year, accounting for nearly 20% of the total number of publications.

Figure 2

As illustrated in Figure 3, while the discovery of iPSC cells in 2006 boosted stem cell research noticeably beginning in 2008, the introduction of CRISPR in 2012 has further catapulted the stem cell field, with combined gene-editing and stem cell publications taking share.

Figure 3

Why Is The Addition of Gene-Editing So Important to Stem Cell Research and Therapy?

Figure 4

Ultimately, CRISPR edited iPSCs should unlock the code to human disease at a cellular level. Three public CRISPR companies are in a good position for the impending stem cell revolution. Editas Medicine (EDIT) signed a stem cell pact with GlaxoSmithKline (GSK) and Biogen (BIIB) for next-gen stem-cell therapies; CRISPR Therapeutics (CRSP) CRSP),+Casebia+Therapeutics+Sign+Commercial+License+Agreement+to+MaxCyte/12664463.html" rel="nofollow">licensed a CRISPR delivery mechanism for blood stem cells and has formed a CRISPR joint venture with Bayer (BAYZF); and Intellia Therapeutics (NTLA) has partnered with biotech giant Novartis NVS to focus on stem-cell based therapies. These companies, among others, finally might unleash the limitless possibilities that stem cells once promised in regenerative medicine, extending human life spans considerably.

Notes

[1] The Weissman Lab at Stanford University first isolated hematopoietic (blood) stem cells from mice bone marrow in 1988; it would be another decade before human stem cells were isolated.

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The Stem Cell Revolution - Seeking Alpha

Evotec in neurology iPSC drug discovery collaboration with stem-cell specialist Censo – FierceBiotech

Evotec has reached a collaboration with Censo Biotechnologies, using the latters patient-derived induced pluripotent stem cells (iPSC) to expand its platform for drug discovery.

Scientists get iPSCs by reprograming human adult somatic cells to make them resemble embryonic stem cells, which can become any cell type of the body. For this alliance, Censo will use its donors sourcing and cell reprograming technologies to create a bespoke library of hundreds of patient-derived iPS cell lines, and Evotec will use them for target validation, drug screening, drug metabolism and pharmacokinetics analyses.

These stem cell-like cells created from people suffering from a disease allow researchers to replicate or mimic the disease ex vivo under lab conditions. This approach is particularly helpful for understanding of the mechanism of diseases such as Parkinsons and Alzheimers. The current contract between Evotec and Censo, with an initial two-year term, will first focus on central nervous system diseases, an area that both companies excel in.

RELATED:Axiogenesis in stem cell collab with ion channel expert Metrion

The Hamburg, Germany-based Evotec has been involved in neurological research for over two decades, having taken part in the discovery process of about a dozen preclinical candidates. The CROs expertise spans some key target classes such as enzymes, GPCRs and ion channels, and in therapeutic areas of neurodegenerative diseases, neuroinflammation, among others.

Censo is the product of a 2016 merger between Roslin Cellab and Roslin Cell Sciencestwo spinouts from the Roslin Institute both focused on using stem cells for drug discovery. Operating from its Edinburgh and Cambridge facilities in the U.K., Censo can generate iPSCs from large cohorts of samples for drug researches, and usually partner with other companies for assay development.

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Evotec in neurology iPSC drug discovery collaboration with stem-cell specialist Censo - FierceBiotech