Category Archives: Somatic Stem Cells

Going Gray Too Soon? Scientists Say It Really May Be Due to Stress – Genetic Engineering & Biotechnology News

Stress has long been anecdotally linked with prematurely graying hair. Its said, for example, that when Marie Antoinette was captured during the French Revolution, her hair turned white overnight. Anecdote this may be, but an international research team led by Harvard University scientists has now discovered how stress may, in fact, cause hair to gray. Their studies in mice and laboratory-grown cells showed that stress activates noradrenaline-releasing sympathetic nerves that are part of the fight-or-flight response, which in turn causes permanent damage to pigment-regenerating stem cells in hair follicles.

Everyone has an anecdote to share about how stress affects their body, particularly in their skin and hairthe only tissues we can see from the outside, said Ya-Chieh Hsu, PhD, the Alvin and Esta Star Associate Professor of Stem Cell and Regenerative Biology at Harvard. We wanted to understand if this connection is true, and if so, how stress leads to changes in diverse tissues. Hair pigmentation is such an accessible and tractable system to start withand besides, we were genuinely curious to see if stress indeed leads to hair graying. Hsu is senior author of the teams paper, which is published in Nature, and titled, Hyperactivation of sympathetic nerves drives depletion of melanocyte stem cells.

Empirical as well as anecdotal evidence has linked stress with accelerated hair graying, which is the formation of hairs with no pigment, the authors stated. In recent history, for example, John McCain experienced severe injuries as a prisoner of war during the Vietnam War and lost color in his hair. However, the scientists acknowledged, despite this type of evidence, so far there has been little scientific validation of this link whether stressors are the causal factors, and whether stress-related changes occur at he level of somatic stem cells, remain poorly understood.

Hair follicles that produce new hairs cycle between phases of growth (anagen), degeneration (catagen), and rest (telogen). The hair follicle contains two types of stem cell, hair follicle stem cells (HFSCs), and pigment-forming melanocyte stem cells (MeSCs). For much of the cycle these stem cells are dormant, but they are activated during early anagen to form new pigmented hairs. The MeSCs act as a reservoir of pigment-producing cells, so when hair regenerates, some of the MeSC stem cells convert into pigment-producing cells that color the hair. differentiated melanocytes synthesize melanin to color the newly regenerated hair from the root, the scientists stated.

Stress affects the whole body, so to investigate any link between stress and hair graying, the authors first had to try to identify which body system was responsible. Their work involved a series of studies, starting with whole-body response and progressively zooming into individual organ systems, cell-to-cell interaction and then down to molecular dynamics. A range of research tools were employed, including methods to manipulate organs, nerves, and cell receptors.

The investigators initial hypothesis was that stress might cause an immune attack on pigment-producing cells. However, their experiments showed that mice lacking immune cells still showed hair graying. The team then looked for a link between stress, graying hair and cortisol, but this also proved negative. Using a combination of adrenalectomy, denervation, chemogenetics, cell ablation and knockout of the adrenergic receptor specifically in melanocyte stem cells, we find that the stress-induced loss of melanocyte stem cells is independent of immune attack or adrenal stress hormone, they noted. Stress always elevates levels of the hormone cortisol in the body, so we thought that cortisol might play a role, Hsu said. But surprisingly, when we removed the adrenal gland from the mice so that they couldnt produce cortisol-like hormones, their hair still turned gray under stress.

After systematically eliminating different possibilities, the researchers honed in on the sympathetic nerve system, which is responsible for the bodys fight-or-flight response. Sympathetic nerves branch out into each hair follicle on the skin. The teams experiments showed that stress causes these nerves to release noradrenaline, which gets taken up by the nearby MeSC pigment-regenerating stem cells.

This image illustrates the elaborate sympathetic innervation (green) around melanocyte stem cells (red). Acute stress induces hyperactivation of the sympathetic nervous system to release large amount of norepinephrine, a neurotransmitter. Norepinephrine drives rapid depletion of melanocyte stem cells and hair graying. [Bing Zhang and Ya-Chieh Hsu]The noradrenaline then triggers excessive activation of the stem cells, which effectively all convert into pigment-producing cells, prematurely depleting the reservoir. Under conditions of stress, the activation of these sympathetic nerves leads to burst release of the neurotransmitter noradrenaline (also known as norepinephrine), the team explained. This causes quiescent melanocyte stem cells to proliferate rapidly, and is followed by their differentiation, migration and permanent depletion from the niche.

We were conducting a study on pain using black C57 mice, a dark-furred laboratory strain, explained co-author Thiago Mattar Cunha, PhD, a researcher affiliated with the Center for Research on Inflammatory Diseases (CRID), a Research, Innovation and Dissemination Center (RIDC) funded by So Paulo Research Foundation (FAPESP) and hosted by the University of So Paulos Ribeiro Preto Medical School (FMRP-USP) in So Paulo State, Brazil. In this model, we administered a substance called resiniferatoxin to activate a receptor expressed by sensory nerve fibers and induce intense pain. Some four weeks after systemic injection of the toxin, a PhD student observed that the animals fur had turned completely white.

After repeated tests the CRID researchers concluded that the phenomenon was due to the application of resiniferatoxin, a naturally occurring chemical found in resin spurge (Euphorbia resinifera), a cactus-like plant native to Morocco. We set out to check the hypothesis that the loss of fur color resulted from pain-induced stress, Cunha said. We designed a very simple experiment to see if the phenomenon was dependent on activation of sympathetic nerve fibers.

After injecting resiniferatoxin into the mice, the animals were treated using guanethidine, an anti-hypertensive that can inhibit neurotransmission via sympathetic fibers. We observed that the process of fur color loss was blocked by the treatment, Cunha said. In another experiment, neurotransmission was interrupted by the surgical removal of sympathetic fibers. In this case, too, fur color was not lost in the weeks following pain induction.

This image illustrates the elaborate sympathetic innervation (magenta) around melanocyte stem cells (yellow). Acute stress induces hyperactivation of the sympathetic nervous system to release large amount of norepinephrine, a neurotransmitter. Norepinephrine drives rapid depletion of melanocyte stem cells and hair graying. [Bing Zhang and Ya-Chieh Hsu]These and other experiments conducted by our group demonstrated the participation of sympathetic innervation in achromotrichia and confirmed that pain is a powerful stressor in this model. But it remained to detail the mechanisms involved, Cunha noted. We used various methodologies to show that intense sympathetic activity speeds up differentiation significantly. In our model, therefore, pain accelerated the aging of the stem cells that produce melanin.

Hsu added, When we started to study this, I expected that stress was bad for the bodybut the detrimental impact of stress that we discovered was beyond what I imagined. After just a few days, all of the pigment-regenerating stem cells were lost. Once theyre gone, you cant regenerate pigment anymore. The damage is permanent.

Cunha noted, For the longest time its been said that stress makes the hair turn white but until now there was no scientific basis for this belief. Our study proved that the phenomenon does indeed occur, and we identified the mechanisms involved. In addition, we discovered a way of interrupting the process of hair color loss due to stress.

The researchers used RNA sequencing to explore the mechanisms that promote melanocyte stem cell differentiation, by comparing the gene expression profiles of mice that received the injection of resiniferatoxin, and developed pain, stress and fur color loss, with those of control mice injected with a placebo.We looked for genes whose expression was most altered after stress induction, and one caught our attention: the gene that encodes a protein called CDK [cyclin-dependent kinase]. This is an enzyme that participates in cell cycle regulation, Cunha said. When the researchers repeated the pain induction procedure and treated the mice with a CDK inhibitor, they found that melanocyte stem cell differentiation was prevented, as was fur color loss. This finding shows that CDK participates in the process and could, therefore, be a therapeutic target, Cunha said. Its too soon to know whether it will actually become a target someday in clinical practice, but its worth exploring further.

The researchers experiments demonstrated that when the sympathetic system is robustly activated, the fibers that innervate hair follicle bulbs release noradrenaline very near the melanocyte stem cells. We showed that melanocyte stem cells express the protein ADRB2 [2-adrenergic receptor], which is activated by noradrenaline, and we discovered that the stem cells differentiate when this receptor is activated by noradrenaline, Cunha said. To confirm the finding, the researchers repeated their tests using mice that had been genetically modified, so as not to express ADRB2. As suspected, the fur of these animals did not turn white after they were injected with resiniferatoxin. In another test, we injected noradrenaline directly into the skin of the mouse. As a result, the fur around the site of the injection turned white, Cunha said.

In a final set of studies, the group showed that cultured primary human melanocytes (melanin-producing cells obtained directly from the skin of a volunteer) treated with noradrenaline showed increased expression of CDK , which was similar to the findings in mice.

According to Cunha, the researchers do not yet know if there will be future aesthetic applications for their findings, such as the development of a drug that could stop us growing gray as we age. It would be necessary to see if a CDK inhibitor has side-effects, and if so whether they would be outweighed by the aesthetic benefit.

Co-author Isaac Chiu, PhD, assistant professor of immunobiology at Harvard Medical School, studies the interplay between nervous and immune systems. He said, we know that peripheral neurons powerfully regulate organ function, blood vessels, and immunity, but less is known about how they regulate stem cells. With this study, we now know that neurons can control stem cells and their function, and can explain how they interact at the cellular and molecular level to link stress with hair graying.

The researchers suggest that their results underscore the negative side effects of an otherwise protective evolutionary response. Acute stress, particularly the fight-or-flight response, has been traditionally viewed to be beneficial for an animals survival. But in this case, acute stress causes permanent depletion of stem cells, said postdoctoral fellow Bing Zhang, first author of the study. To go from the highest level to the smallest detail, we collaborated with many scientists across a wide range of disciplines, using a combination of different approaches to solve a very fundamental biological question.

The scientists also acknowledged that the reason for any interaction between nerves and MeSCs isnt known. The connection between the nervous system and pigment-producing cells is probably conserved during evolution they suggested. Squid, cuttlefish, and octopus are cephalopods that can rapidly change color for camouflage or to communicate. Their nervous system controls pigment-producing chromatophore cells, allowing very fast changes in color in response to threats or predators. Therefore, an attractive hypothesis is that sympathetic nerves might modulate MeSC activity, melanocyte migration or pigment production in situations independent of the hair cyclefor example, under bright sunlight or UV irradiation, the team suggested. Under extreme stress, however, hyperactivation of neuronal activities overstimulates the pathway, which drives the depletion of MeSCs.

The findings could help to provide new insights into the broader effects of stress on various organs and tissues, which could ultimately lead to new approaches to modifying or blocking the damaging effects of stress. By understanding precisely how stress affects stem cells that regenerate pigment, weve laid the groundwork for understanding how stress affects other tissues and organs in the body, Hsu said. Understanding how our tissues change under stress is the first critical step towards eventual treatment that can halt or revert the detrimental impact of stress. We still have a lot to learn in this area.

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Going Gray Too Soon? Scientists Say It Really May Be Due to Stress - Genetic Engineering & Biotechnology News

Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – Business Wire

SAN DIEGO--(BUSINESS WIRE)--Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About AlleleAllele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

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Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - Business Wire

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

Stem Cells Market- What Are The Main Factors That Contributing Towards Industry Growth? – Industry Mirror

Stem Cells Market AnalysisAccording to Market Research, the Global Stem Cells Market was valued at USD 5.88 Billion in 2018 and is expected to witness a growth of 10.32% from 2019-2026 and reach USD 12.96 Billion by 2026.

What is Stem Cells Market?Stem cellscan be defined as unspecialized cells that develop into the specialized cells and make up different types of tissue in the human body. Since stem cells are unspecialized type of cells and are capable of renewing themselves through cell division. Stem cells can be Pluripotent as well as Multipotent.

Pluripotent stem cells are stem cells usually found in embryos which give rise to all the cells found in the human body, while multipotent stem cells, which are found in adults or in babies umbilical cords, have a more restricted ability. Their development is limited to cells that form the organ system that they are originated from. When a stem cell undergoes division, each new cell possess a potential either to remain a stem cell or develop into another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

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Stem Cells Market OutlookStem cell research is considered as one of the most intriguing areas of contemporary biology, but, as with many expanding fields of scientific inquiry, research on stem cells stimulates scientific queries as rapidly as it produces new discoveries. Until recently, scientists used to primarily work with two types of stem cells from animals and humans: embryonic stem cells and non-embryonic somatic or adult stem cells.

Since the advent of stem cells, one of the crucial benefits of stem cell research is the accessibility of cell lines and that they can be acquired ethically. The demands for pluripotent stem cells are increasing owing to the fact that it differentiates in various cell types in the human body. Pluripotent stem cells tend to have various applications in the medical treatment. Growing awareness regarding the stem cells and establishment of stem cell banks is expected to fuel the market growth rate.

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Ethical issues related to pluripotent stem cells could hamper the growth of stems cells in the global market as research with these cells require disrupting an artificially-fertilized embryo at the 5-14 day stage. Another factor which is limiting the growth of stem cells market is unknown long-term consequences.

Global Stem Cells Market SegmentationThe Global Stem Cells Market is classified on the basis of Product, Treatment Type, Therapeutic Application and Region. The gist of breaking down the market into various segments is to gather the information about various aspects of the market.

On the basis of Products, the market is bifurcated on the basis of Adult Stem Cells, Human Embryonic Cells, and Induced Pluripotent Stem Cell. Adult stem cells accounts for a major share in the global stem cells market. Even though embryonic stem cells have a wide range of applications, the market growth rate for this sub-segment is substantial owing to the ethical issues faced by this sub-segment in the global market.

In terms of Therapeutic Application, the market study encompasses various aspects such ca Regenerative Medicine, Neurological Disorders, Orthopedic Treatments, Oncology Disorders, Diabetes, Injuries & Wounds and Cardiovascular Disorders. Growing awareness regarding regenerative medicine is expected to make this sub-segment hold a potential market share globally. Growing healthcare expenditure and presence of major industry players makes North America hold major share in the global market.

Stem Cells Market Competitive LandscapeThe Stem Cells Market study report offers a valuable insight with an emphasis on global market including some of the major players such asBioTime Inc., Cytori Therapeutics, Inc., STEMCELL Technologies Inc., Astellas Pharma Inc., U.S. Stem Cell, Inc., Osiris Therapeutics, Inc., Takara Bio Inc., Caladrius Biosciences, Inc., Cellular Engineering Technologies Inc., and BrainStorm Cell Therapeutics Inc. Our market analysis also entails a section solely dedicated for such major players wherein our analysts provide an insight to the financial statements of all the major players, along with its product benchmarking and SWOT analysis. The competitive landscape section also includes key development strategies, market share and market ranking analysis of the above mentioned players globally.

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Analyst View:As per our sources following trends were observed in terms of most popular sources of stem cells:

Stem cells from adult bone marrow were observed to be the most popular source. Scope of stem cell therapy is increasing with growing number of applications. Clinical research has advanced to a great magnitude towards preventing, identifying and handling devastating diseases. Various applications of stem cells in regeneration such as Cardiac Regeneration, Hepatic Regeneration, Regeneration of Neural Tissue, etc. have come up lately. This suggests that the market for stem cells will grow significantly over the forecast period.

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it – FOX 10 News Phoenix

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it - FOX 10 News Phoenix

Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key – Market Research Sheets

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

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The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

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The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key - Market Research Sheets

Cell Therapy Industry Applications 2019-Size by Type (Allogenic Therapies), by Technique (Stem Cell Therapy), Global Market Growth by Demand Analysis…

TheGlobal Cell Therapy Marketwas estimated to be valued at USD XX million in 2018 and is projected to reach USD XX million by 2026, at a CAGR of XX% during 2019 to 2026.

Cell therapy involves the administration of somatic cell preparations for the treatment of diseases or traumatic damages. The objective of this study is to provide long term treatment through a single injection of therapeutic cells.

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Growing aging patient population, the rise in cell therapy transplantations globally, and rising disease awareness drive the growth of the market. However, stringent regulatory policies may restrain growth of the market in the forecast period.

The Global Cell Therapy Industry is primarily segmented based on different type, technique, cell source, technology, end users and region

On the basis of type, the market is split into:

On the basis of technique, the market is split into:

On the basis of cell source, the market is split into:

On the basis of technology, the market is split into:

On the basis of end user, the market is split into:

Moreover, the market is classified based on regions and countries as follows:

Key players profiled in the report includes:

These organizations are focusing on growth strategies, such as new product launches, expansions, acquisitions, and agreements & partnerships to expand their operations across the globe.

GlobalCell TherapyIndustry isspread across 121 pages, profiling 10 companies and supported with tables and figures. Inquire more or share a questions if any before the purchase on this report @

What you can expect from our report: Total Addressable Market [ Present Market Size forecasted to 2026 with CAGR ] Regional level split [North America, Europe, Asia Pacific, South America, Middle East & Africa] Country wise Market Size Split [Important countries with major market share] Market Size Breakdown by Product/ ServiceTypes [ ] Market Size by Application/Industry verticals/ End Users [ ] Market Share and Revenue/Sales of 10-15 Leading Players in the Market Production Capacity of Leading Players whenever applicable Market Trends Emerging Technologies/products/start-ups, PESTEL Analysis, SWOT Analysis, Porters Five Forces, etc. Pricing Trend Analysis Average Pricing across regions Brandwise Ranking of Major Market Players globally

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Research Methodology:

The market is derived through extensive use of secondary, primary, in-house research follows by expert validation and third party perspective, such as, analyst reports of investment banks. The secondary research is the primary base of our study wherein we conducted extensive data mining, referring to verified data products, such as, white papers, government and regulatory published articles, technical journals, trade magazines, and paid data products.

For forecasting, regional demand & supply factors, recent investments, market dynamics including technical growth scenario, consumer behavior, and end use trends and dynamics, and production capacity were taken into consideration. Different weightages have been assigned to these parameters and quantified their market impacts using the weighted average analysis to derive the market growth rate.

The market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs), which typically include:

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Table of Content

1. Introduction2. Research Methodology3. Executive Summary4. Global Cell Therapy Market Overview5. Global Cell Therapy Market, by Type6. Global Cell Therapy Market, by Technique7. Global Cell Therapy Market, by Cell Source8. Global Cell Therapy Market, by Technology9. Global Cell Therapy Market, by End Users10. Global Cell Therapy Market by Region11. Competitive Landscape12. Company Profiles13. Cell Therapy Manufacturing Cost Analysis14. Key InsightsEnd of the reportDisclaimer

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Cell Therapy Industry Applications 2019-Size by Type (Allogenic Therapies), by Technique (Stem Cell Therapy), Global Market Growth by Demand Analysis...

Stem Cell Therapies Market research Likely to Emerge over a Period of 2015-2025 –

VALLEY COTTAGE, N.Y. Stem cells are undifferentiated biological cells, and having remarkable potential to divide into any kind of other cells. When a stem cell divides, each new cell will be a new stem cell or it will be like another cell which is having specific function such as a muscle cell, a red blood cell, brain cell and some other cells.

There are two types of stem cells

Stem cells harvested from umbilical cord blood just after birth. And this cells can be stored in specific conditions. Stem cells also can be harvest from bone marrow, adipose tissue.

Embryonic cells can differentiate into ectoderm, endoderm and mesoderm in developing stage. Stem cells used in the therapies and surgeries for regeneration of organisms or cells, tissues.

Stem cells are used for the treatment of Gastro intestine diseases, Metabolic diseases, Immune system diseases, Central Nervous System diseases, Cardiovascular diseases, Wounds and injuries, Eye diseases, Musculoskeletal disorders.

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Harvesting of Adult cell is somewhat difficult compare to embryonic cells. Because Adult cells available in the own body and it is somewhat difficult to harvest.

Stem Cell TherapiesMarket: Drivers and Restraints

Technology advancements in healthcare now curing life threatening diseases and giving promising results. Stem Cell Therapies having so many advantages like regenerating the other cells and body organisms. This is the main driver for this market. These therapies are useful in many life threatening treatments. Increasing the prevalence rate of diseases are driven the Stem Cell Therapies market, it is also driven by increasing technology advancements in healthcare. Technological advancements in healthcare now saving the population from life threatening complications.

Increasing funding from government, private organizations and increasing the Companies focus onStem cell therapiesare also driven this market

However, Collecting the Embryonic Stem cells are easy but Collecting Adult Stem cell or Somatic Stem cells are difficult and also we have to take more precautions for storing the collected stem cells.

Preview Analysis of Stem Cell Therapies Market: Global Industry Analysis and Opportunity Assessment 2015 2025:

Stem Cell TherapiesMarket: Segmentation

Stem Cell Therapies are segmented into following types

Based on treatment:

Based on application:

Based on End User:

Stem Cell TherapiesMarket: Overview

With rapid technological advantage in healthcare and its promising results, the use of Stem Cell Therapies will increase and the market is expected to have a double digit growth in the forecast period (2015-2025).

Stem Cell TherapiesMarket: Region- wise Outlook

Depending on geographic regions, the global Stem Cell Therapies market is segmented into seven key regions: North America, South America, Eastern Europe, Western Europe, Asia Pacific excluding Japan, Japan and Middle East & Africa.

The use of Stem Cell Therapies is high in North America because it is highly developed region, having good technological advancements in healthcare setup and people are having good awareness about health care. In Asia pacific region china and India also having rapid growth in health care set up. Europe also having good growth in this market.

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Stem Cell TherapiesMarket: Key Players

Some of the key players in this market are

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Stem Cell Therapies Market research Likely to Emerge over a Period of 2015-2025 -

Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals – Proactive Investors USA & Canada

CEO Vered Caplan says the move would enable the development and delivery of cell and gene therapies onsite at HYGEIA's hospitals in Greece

Inc (), a developer of advanced cell therapies, revealed Friday that it struck a strategic partnership agreement between the Theracell joint venture and the large HYGEIA Group which runs three hospitals in Greece.

In a statement, the Germantown, Maryland-based company said that under the terms of the agreement, the joint venture will implement point-of-care cell therapy platform for clinical development and commercialization of cell and gene therapies within the HYGEIA Groups network of hospitals in Greece.

and TheraCell Advanced Biotechnology earlier formed a joint venture to advance point-of-care platform in Greece, the Balkan region and some Middle Eastern countries.

The point-of-care platform is designed to collect, process and supply cells within the patient care setting for various treatments.

The main goal is to reduce the cost and complexity of supplying cell and gene therapies, said , as well as boostquality by integrating automated processing units and proprietary technologies.

Significantly, HYGEIA is the first hospital network in the region to implement Orgenesis point-of-care cell therapy platform. The partnership aims to provide the HYGEIA Group with resources to advance clinical development and deliver personalized, advanced therapies across its network for a range of diseases in oncology, hematology, orthopedics, nephrology, dermatology and diabetes.

This partnership with the HYGEIA Group further validates the significant value proposition of our point-of-care platform, as it enables the development and delivery of cell and gene therapies onsite at hospitals, said Orgenesis CEO Vered Caplan.

We believe this platform has the potential to transform the cell and gene therapy market, by bringing life-saving therapies to market in a much more time and cost-effective manner, she added.

The Orgenesis boss said Theracell had proved to be an ideal partner with extensive experience and capabilities in autologous cell therapy and regenerative medicine, and strong operations in Greece and relationships in the region.

We are in active discussions to establish PoCare locations and partnerships with hospitals and healthcare networks in other countries and regions across the world, said Caplan.

Greeces HYGEIA Group operates three hospitals with a capacity of 1,261 beds, 52 operating rooms, 19 delivery rooms and 10 intensive care units.

HYGEIA Group CEO Andreas Kartapanis said thanks to the partnership with Theracell and Orgenesis it would be the first hospital network in Greece to provide advanced cell and gene therapies.

We believe this partnership will provide us a strong competitive advantage in this rapidly developing field. More importantly, this partnership will benefit patients that will now have greater access to these important therapies, said Kartapanis.

For the fiscal third quarter ended September 30, Orgenesis generated meaningful revenue, over $1 million, through its rapidly advancing point-of-care cellular therapy platform.

Meanwhile, TheraCell has experience in the isolation, processing and application of adipose-derived stem cells, as well as somatic cells. It has developed a patented platform for tissue engineering and cell therapies in the areas of dermatology, articular cartilage defects, and chronic kidney injury.

Contact Uttara Choudhury at[emailprotected]

Follow her onTwitter:@UttaraProactive

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Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals - Proactive Investors USA & Canada

Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia – BioSpace

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the first patient in the global Phase 2 registration-enabling study of RP-L102 Process B received investigational therapy. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA).

The initiation of Rockets first Phase 2 trial is an important milestone for the company as well as patients throughout the world battling FA, said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development of Rocket. With the recent feedback received from the FDA and EMA of MMC-resistance as the primary endpoint, we are optimistic about the prospect of benefiting patients and, if the data are positive, working towards BLA and MAA submissions.

The registrational package will include twelve patients from the U.S. and EU, two from the U.S. Phase 1 study and 10 additional patients from the global Phase 2 study (NCT04069533). Patients will receive a single intravenous infusion of RP-L102 that utilizes fresh cells and Process B which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product. Improved mitomycin-C (MMC) resistance in bone marrow colony forming (progenitor) cells is the primary endpoint, and may also serve as a surrogate endpoint for accelerated approval. Additional outcome measures include stability or increase in blood counts with no significant worsening in anemia, neutropenia or thrombocytopenia and peripheral blood and bone marrow genetic correction, as demonstrated by progressive increases in vector copy number (VCN) over the months subsequent to infusion.

Lucile Packard Childrens Hospital Stanford and Hospital Infantil Universitario Nio Jess are serving as the lead clinical sites and University of Minnesota is conducting centralized evaluation of bone marrow MMC-resistance and engaging in advisory activities for the global trial of RP-L102. RP-L102 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER), Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD) and Fundacion para la Investigacion Biomedica Hospital Infantil Universitario Nio Jesus (FIB-HIUNJ).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of solid tumors, particularly upper aerodigestive tract squamous cell carcinomas. Approximately 60-70% of patients with FA have a FANCA gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. These assays can further differentiate FA patients from mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patients blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients contending with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia - BioSpace