Category Archives: Stem Cell Medicine


Cellectis stem cells today proudly announces the launch of the world’s very first human iPS cell-derived hepatocyte …

GOTHENBURG, Sweden--(BUSINESSWIRE)--

Regulatory News:

Cellectis stem cells, a Business Unit of Cellectis Group (Alternext: ALCLS), a premier provider of stem cell derived products and technologies, today announces the launch of a human iPS derived hepatocyte product, hiPS-HEPTM.

The hiPS-HEPTM demonstrate high reproducibility, homogeneity and a long life span of stable CYP activity, making them the ideal platform for various in vitro applications including drug discovery, toxicity testing and vaccine development. The hiPS-HEP are human hepatocyte-like cells derived from human induced Pluripotent Stem (iPS) cells under strict quality controlled and ethically approved procedures.

"Due to their high relevance in various industrial applications it makes the hiPS-HEP a really promising system for research and development," said Johan Hyllner, CSO of Cellectis stem cells. "The pharmaceutical industry has a great need for better and more clinically relevant models early in the drug development process to predict hepatotoxicity, find new drug targets and develop new vaccines."

"This novel product is the fruition of Cellectis strategy to become the global market leader for stem cell-based in vitro models and related technologies. It illustrates our ambitions and the momentum of our future development in this field," said Andr Choulika, Chairman and CEO of Cellectis.

About Cellectis stem cells:

Cellectis stem cells, is a business unit within the Cellectis group and is a global leader in stem cell technology. Cellectis stem cells, created in November 2011 from Cellartis AB and Ectycell SAS, possesses broad expertise in pluripotent stem cells, including iPS cell technology, genetic engineering and specialised cells. Cellectis stem cells is developing stem cell derived products and related services for drug discovery, toxicity testing and regenerative medicine applications.

For more information visit http://www.cellartis.com and http://www.cellectis.com

About Cellectis

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Cellectis stem cells today proudly announces the launch of the world’s very first human iPS cell-derived hepatocyte ...

Stem cell collaboration could set stage for company’s growth

A stem cell breakthrough at UCLA could mark a big step for a biopharmaceutical company to use its proprietary technology to forge partnerships with pharmaceutical companies and other research institutions.

Fibrocell Sciences technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen. In a research collaboration with the company, UCLA used the technology to isolate, identify and increase the number of different skin cell types, which lead to two rare adult stem cell-like subpopulations being identified in adult human skin SSEA3-expressing regeneration-associated cells associated with skin regeneration after injuries and mesenchymal adult stem cells.

The findings could have broad applications for personalized medicine. Currently, adult stem cells are derived from adipose tissue and bone marrow. Using mesenchymal stem cells would be less invasive and could be more efficient. Mesenchymal stem cells are being used in research to develop osteoblasts, or bone cells; chondrocytes, or cartilage cells; and adipocytes, or fat cells.

David Pernock, the chairman and CEO of Fibrocell, said the move could mark a significant step in the companys growth.

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Pernock added: Once we have shown we can produce these stem cells in meaningful quantities safely and efficiently, I think well be in a position where companies would want to partner with us to develop them for a variety of indications.

In addition to collaborations, the company has been developing its own therapeutics.

The company launched its first U.S. Food and Drug Administration-approved therapy Laviv last year. The therapy uses individuals fibroblast cells to reduce nasolabial fold wrinkles, folds on both sides of the face that start from the outer corners of the nose down to the corners of the mouth. It is also advancing its acne therapy through phase 3 clinical trials and its burn scar therapy through phase 2 trials.

Pernock joined the company two years ago from GlaxoSmithKline. He said the developments under way at the company indicate it is growing and expects to add engineering staff to its Exton, Pennsylvania office later this year.

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Stem cell collaboration could set stage for company’s growth

Harvard Stem Cell Institute Sees Growth

At its founding eight years ago, the Harvard Stem Cell Institute had fewer than ten principal faculty members, according to Benjamin D. Humphreys, co-director of the HSCI Kidney Program. Today, that number has ballooned to more than 80.

In the past decade, Harvard has increasingly poured resources into groundbreaking research in one of the largest collections of stem cell research labs in the country.

According to HSCI co-director Douglas A. Melton, a professor in the stem cell and regenerative biology department, there are more than 800 Harvard affiliates in stem cell science scattered throughout roughly 80 laboratories. The largest concentration of stem cell researchers are located in Harvards Sherman Fairchild Building, which reopened in August of 2011 after it underwent a two-year demolition and reconstruction project to accommodate the stem cell and regenerative biology department.

In the past decade, Harvard has focused on centralizing this research with the creation of HSCI and the stem cell and regenerative biology department.

HSCI consists of scientists and practitioners interested in stem cell research from all over the Harvard community, including the Faculty of Arts and Sciences, the medical school, and 11 teaching hospitals and research institutions including the Childrens Hospital Boston and the Massachusetts General Hospital.

So far, HSCI has given out more than $100 million to its researchers, according to Humphreys.

"[Harvard has] definitely made a tangible commitment to stem cell research," Humphreys said. "The results are that we are leaders in certain areascertainly I can speak of the kidneynot even just in the U.S., but worldwide in terms of stem cell research in the kidney."

With important potential applications such as the generation of cells and tissues that could be used for cell-based therapies, stem cells are at the forefront of scientific research. Stem cells, which can differentiate into specific cell types, offer the possibility of a renewable source of replacement cells and tissues to treat some of the most serious diseases.

"What were doing at the HSCI Kidney Group is working collaboratively to identify new therapeutic strategies that will help slow disease progression," said Humphreys.

Still, Humphreys added that much more research is necessary before scientists can use stem cells to their fullest potential.

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Harvard Stem Cell Institute Sees Growth

MissionIR News – VistaGen Therapeutics to Speed Development of Drug Screening and Cell Therapy for Immune System …

Atlanta, GA (PRWEB) April 17, 2012

MissionIR would like to highlight VistaGen Therapeutics, Inc. (OTCBB: VSTA), focused on applying stem cell technology for drug rescue and cell therapy. Drug rescue combines human stem cell technology with modern medicinal chemistry to generate new chemical variants ("drug rescue variants") of promising drug candidates that have been discontinued during preclinical development ("put on the shelf") due to heart or liver safety concerns.

In the companys news yesterday,

VistaGen Therapeutics announced it has licensed breakthrough stem cell culture technology from the McEwen Centre for Regenerative Medicine located at the University Health Network (UHN) in Toronto, Canada.

VistaGen will be utilizing the licensed technology to develop hematopoietic precursor stem cells from human pluripotent stem cells, with the goal of developing drug screening and cell therapy applications for human blood system disorders. The breakthrough technology is included in a new United States patent application.

Hematopoietic precursor stem cells give rise to all red and white blood cells and platelets in the body. VistaGen will use the UHN invention to improve the cell culture methods used to efficiently produce hematopoietic stem cell populations.

This technology dramatically advances our ability to produce and purify this important blood stem cell precursor for both in vitro drug screening and in vivo cell therapy applications, said H. Ralph Snodgrass, PhD, VistaGens President and Chief Scientific Officer.

In addition to defining new cell culture methods for our use, the technology describes the surface characteristics of stem cell-derived adult hematopoietic stem cells. Most groups study embryonic blood development from stem cells, but, for the first time, we are able to not only purify the stem cell-derived precursor of all adult hematopoietic cells, but also pinpoint the precise timing when adult blood cell differentiation takes place in these cultures, Snodgrass added. It is our belief that these early cells will be the precursors of the ultimate adult, bone marrow-repopulating hematopoietic stem cells.

Bone marrow-derived hematopoietic stem cells are able to repopulate the blood and immune system when transplanted into patients prepared for bone marrow transplantation. These cells have important potential therapeutic applications for the restoration of healthy blood and immune systems in individuals undergoing transplantation therapies for cancer, organ grafts, HIV infections, or for acquired or genetic blood and immune deficiencies.

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MissionIR News - VistaGen Therapeutics to Speed Development of Drug Screening and Cell Therapy for Immune System ...

New Data Show BioCision’s CoolCell Family of Products Outperform Traditional Cryopreservation Methods

MILL VALLEY, Calif.--(BUSINESS WIRE)--

BioCision LLC today released new data demonstrating that the companys CoolCell technology resulted in a 33 percent increase in human embryonic stem cell (hESC) viability when compared to common freezing methods, such as styrofoam boxes, paper towel wraps and alcohol-based methods.

Based on thermo-conductive principles and developed through precision-engineering, CoolCell is a rate-controlled cryopreservation system that ensures uniform and reproducible freezing rates for biological samples, including primary cells, cell lines, stem cells, yeast and other cell types.

Addressing the lack of standardization in common sample handling, processing and viable long-term storage has emerged as one of the greatest challenges facing biobanks, diagnostics companies and researchers today, said Rolf Ehrhardt, CEO, BioCision. Scientists know that error is inherent in sample handling and can be the difference between viable and non-viable cells, particularly when handling embryonic stem cells that are extremely fragile following thawing.1

Researchers set out to compare cell recovery rates post-thaw using the CoolCell method of cryopreservation and three commonly used methods with two human Embryonic Stem Cell (hESC) lines, RC-7 and RC-10. The study concluded that:

The study was conducted in collaboration with scientists at Edinburgh-based Roslin Cellab, a stem cell technology company with expertise in the stem cell culture and regenerative medicine field, and sister company to Roslin Cells, a leading European and UK provider of clinical grade human embryonic stem cells.

The results of our study show that the CoolCell technology clearly outperforms all other methods and provides excellent efficiency and reproducibility for stem cell cryopreservation and recovery, said Aidan Courtney, Director at Roslin Cellab.

About CoolCell

CoolCell alcohol-free cell cryopreservation systems provide highly reproducible controlled-rate freezing for eukaryotic cells, including primary cells, cell lines, stem cells, PBMC, yeast and other types of cells. Used worldwide in academic, research and clinical laboratories, CoolCell is the standard for passive controlled-rate cell cryopreservation. The CoolCell technology - using novel thermo-conductive alloy and highly insulative materials - ensures precise heat removal from each tube and standardizes the freezing experience of all cryogenic tubes in a -80C freezer.

Benefits include:

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New Data Show BioCision’s CoolCell Family of Products Outperform Traditional Cryopreservation Methods

Somatic stem cells obtained from skin cells for first time ever

"Our research shows that reprogramming somatic cells does not require passing through a pluripotent stage," explains Schler. "Thanks to this new approach, tissue regeneration is becoming a more streamlined - and safer - process."

Up until now, pluripotent stem cells were considered the 'be-all and end-all' of stem cell science. Historically, researchers have obtained these 'jack-of-all-trades' cells from fully differentiated somatic cells. Given the proper environmental cues, pluripotent stem cells are capable of differentiating into every type of cell in the body, but their pluripotency also holds certain disadvantages, which preclude their widespread application in medicine. According to Schler, "pluripotent stem cells exhibit such a high degree of plasticity that under the wrong circumstances they may form tumours instead of regenerating a tissue or an organ." Schler's somatic stem cells offer a way out of this dilemma: they are 'only' multipotent, which means that they cannot give rise to all cell types but merely to a select subset of them - in this case, a type of cell found in neural tissue - a property, which affords them an edge in terms of their therapeutic potential.

To allow them to interconvert somatic cells into somatic stem cells, the Max Planck researchers cleverly combined a number of different growth factors, proteins that guide cellular growth. "One factor in particular, called Brn4, which had never been used before in this type of research, turned out to be a genuine 'captain' who very quickly and efficiently took command of his ship - the skin cell - guiding it in the right direction so that it could be converted into a neuronal somatic stem cell," explains Schler. This interconversion turns out to be even more effective if the cells, stimulated by growth factors and exposed to just the right environmental conditions, divide more frequently. "Gradually, the cells lose their molecular memory that they were once skin cells," explains Schler. It seems that even after only a few cycles of cell division the newly produced neuronal somatic stem cells are practically indistinguishable from stem cells normally found in the tissue.

Schler's findings suggest that these cells hold great long-term medical potential: "The fact that these cells are multipotent dramatically reduces the risk of neoplasm formation, which means that in the not-too-distant future they could be used to regenerate tissues damaged or destroyed by disease or old age; until we get to that point, substantial research efforts will have to be made." So far, insights are based on experiments using murine skin cells; the next steps now are to perform the same experiments using actual human cells. In addition, it is imperative that the stem cells' long-term behaviour is thoroughly characterized to determine whether they retain their stability over long periods of time.

"Our discoveries are a testament to the unparalleled degree of rigor of research conducted here at the Mnster Institute," says Schler. "We should realize that this is our chance to be instrumental in helping shape the future of medicine." At this point, the project is still in its initial, basic science stage although "through systematic, continued development in close collaboration with the pharmaceutical industry, the transition from the basic to the applied sciences could be hugely successful, for this as well as for other, related, future projects," emphasizes Schler. This, then, is the reason why a suitable infrastructure framework must be created now rather than later. "The blueprints for this framework are all prepped and ready to go - all we need now are for the right political measures to be ratified to pave the way towards medical applicability."

More information: Han D.W., Tapia N., Hermann A., Hemmer K., Hing S., Arazo-Bravo M.J., Zaehres H., Frank S., Moritz S., Greber B., Yang J.H., Lee H.T., Schwamborn J.C., Storch A., Schler H.R. (2012) Direct Reprogramming of Fibroblasts into Neural Stem Cells by Defined Factors, Cell Stem Cell, CELL-STEM-CELL-D-11-00679R3

Provided by Max-Planck-Gesellschaft (news : web)

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Somatic stem cells obtained from skin cells for first time ever

Gov. Perry's stem-cell firm draws FDA scrutiny

The U.S. Food and Drug Administration has received a complaint alleging the Houston company involved in Gov. Rick Perry's unregulated adult stem-cell operation is a potential danger to patients and not in compliance with federal law.

In an eight-page letter sent last month, University of Minnesota bioethicist Leigh Turner called on the FDA to investigate Celltex Therapeutics Corp., which banks people's stem cells for future reinjection in the event of disease or injury. Perry was the company's first customer last year.

"It appears their business plan involves injecting or infusing on a for-profit, commercial basis non-FDA-approved adult stem cells into paying customers," Turner wrote in the Feb. 21 letter. "This plan conflicts with FDA regulations governing human stem cells."

An FDA spokeswoman declined comment, but Turner said an agency official told him the matter has been assigned to an investigator and is being taken seriously.

Celltex co-founder David Eller said Tuesday night he is confident the company will "meet all FDA specifications." He emphasized that Celltex doesn't administer stem cells, but stores and processes them at the behest of doctors who later reinject them into patients.

Dr. Stanley Jones, a Houston orthopedic surgeon, injected Perry's stem cells during his back surgery in July.

The plan by Celltex and Perry to make Texas a leader in the therapy have been controversial since details about the governor's procedure became known last summer. The therapy, drawing on the ability of adult stem cells to replenish dying cells, is promising but thought by most medical researchers to need much more clinical study before it is commercialized.

Stem cells are a kind of medicine known as biologics, therapy involving living cells rather than chemicals. Most medical experts say that adult stem-cell therapy involves more than the "minimal manipulation" the agency allows without its oversight because the cells are isolated, cultured in a laboratory and stored for some period of time before being reinjected.

The FDA has recently stepped up enforcement of unregulated adult stem cell activity, though legal experts interviewed last fall by the Chronicle said it was unclear whether the agency would look into Perry's procedure because he seemed fully informed and unharmed by it.

The Texas Medical Board is currently considering a policy that would require providers of stem cells and other experimental drugs to use them only with the permission of independent review committees that assess trials for patient safety. The policy comes up for final approval in April.

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Gov. Perry's stem-cell firm draws FDA scrutiny

New transplant method may allow kidney recipients to live life free of anti-rejection medication

ScienceDaily (Mar. 11, 2012) New ongoing research published March 7 in the journal Science Translational Medicine suggests organ transplant recipients may not require anti-rejection medication in the future thanks to the power of stem cells, which may prove to be able to be manipulated in mismatched kidney donor and recipient pairs to allow for successful transplantation without immunosuppressive drugs. Northwestern Medicine and University of Louisville researchers are partnering on a clinical trial to study the use of donor stem cell infusions that have been specially engineered to "trick" the recipients' immune system into thinking the donated organ is part of the patient's natural self, thus gradually eliminating or reducing the need for anti-rejection medication.

"The preliminary results from this ongoing study are exciting and may have a major impact on organ transplantation in the future," said Joseph Leventhal, MD, PhD, transplant surgeon at Northwestern Memorial Hospital and associate professor of surgery and director of kidney and pancreas transplantation at Northwestern University Feinberg School of Medicine. "With refinement, this approach may prove to be applicable to the majority of patients receiving the full spectrum of solid organ transplants."

Leventhal authored the study along with Suzanne Ildstad, MD, director of the Institute of Cellular Therapeutics at the University of Louisville. It is the first study of its kind where the donor and recipient do not have to be related and do not have to be immunologically matched. Previous studies involving stem cell transplants for organ recipients have included donors and recipients who are siblings and are immunologically identical, something that only occurs in about 25 percent of sibling pairs.

"Being a transplant recipient is not easy. In order to prevent rejection, current transplant recipients must take multiple pills a day for the rest of their lives. These immunosuppressive medications come with serious side effects with prolonged use including high blood pressure, diabetes, infection, heart disease and cancer, as well as direct damaging effects to the organ transplant," said Ildstad. "This new approach would potentially offer a better quality of life and fewer health risks for transplant recipients."

In a standard kidney transplant, the donor agrees to donate their kidney. In the approach being studied, the individual is asked to donate part of their immune system as well. The process begins about one month before the kidney transplant, when bone marrow stem cells are collected from the blood of the kidney donor using a process called apheresis. The donor cells are then sent to the University of Louisville to be processed, where researchers enrich for "facilitating cells" believed to help transplants succeed. During the same time period, the recipient undergoes pre-transplant "conditioning," which includes radiation and chemotherapy to suppress the bone marrow so the donor's stem cells have more space to grow in the recipient's body.

Once the facilitating cell-enriched stem cell product has been prepared, it is transported back to Northwestern, where the recipient undergoes a kidney transplant. The donor stem cells are then transplanted one day later and prompt stem cells to form in the marrow from which other specialized blood cells, like immune cells, develop. The goal is to create an environment where two bone marrow systems exist and function in one person. Following transplantation, the recipient takes anti-rejection drugs which are decreased over time with the goal to stop a year after the transplant.

"This is something I have worked for my entire life," said Ildstad, who pioneered the approach and is known for her discovery of the "facilitating" cell.

Less than two years after her successful kidney transplant, 47-year-old mother and actress Lindsay Porter of Chicago, is living a life that most transplant recipients dream of -- she is currently free of anti-rejection medications and says at times, she has to remind herself that she had a kidney transplant. "I hear about the challenges recipients have to face with their medications and it is significant. It's almost surreal when I think about it because I feel so healthy and normal." Doctors are hopeful that Porter will not need immunosuppressive drugs long-term, given her progress thus far.

In order to qualify for this type of experimental kidney transplant, the donor and recipient pairs must be blood-type compatible and have a negative cross-match, which means that testing has been done to confirm the recipient does not have antibodies in the blood that would cause rejection of the kidney.

The clinical trial is ongoing. Researchers are also planning a second clinical trial, which would offer similar treatment for subjects who have already undergone a living donor kidney transplant.

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New transplant method may allow kidney recipients to live life free of anti-rejection medication

Heart Disease Stem Cell Therapies – Development Must Come From Several Specialties

Editor's Choice Academic Journal Main Category: Heart Disease Also Included In: Cardiovascular / Cardiology;Stem Cell Research Article Date: 09 Mar 2012 - 4:00 PST

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The paper's lead author, Kenneth Chien from Harvard University in the USA explains:

Until now, clinical trials have been based on heart attacks, chronic heart failure as well as dilated cardiomyopathy, but regardless of the fact that regenerative therapies that are based on various non-cardiac cell types seem to be safe, their efficacy has not yet been tested in a clinical trial.

However, possible new targets and treatment strategies are now emerging due to recent progress in cardiac stem cell research and regenerative biology.

Scientists used to think that the heart only has a minimal capacity for self-renewal and saw no prospect in reversing the loss of healthy heart muscle and function. This perception has been altered because of recent findings, such as the discovery of several distinct embryonic progenitor cell types of which some are found in the heart.

A certain number of these cells can be activated in people with cardiac injuries and are now targeted by scientists to develop novel cardiac regenerative therapeutics either by delivery of the cells, or by new methods that activate expansion and conversion of functioning heart cells.

For instance, clinical studies conducted a short while ago demonstrated that scar formation following a heart attack can be reduced by taking cells from the patient's own heart tissue. Even though it remains uncertain whether the delivered cells are indeed stem cells, these studies nevertheless demonstrate that this is a small, educational step towards the goal of utilizing the heart's potential for self-healing.

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Heart Disease Stem Cell Therapies - Development Must Come From Several Specialties

Fly research gives insight into human stem cell development and cancer

Public release date: 8-Mar-2012 [ | E-mail | Share ]

Contact: Phyllis Edelman pedelman@genetics-gsa.org 301-351-0896 Genetics Society of America

CHICAGO, IL March 8, 2012 Stem cells provide a recurring topic among the scientific presentations at the Genetics Society of America's 53rd Annual Drosophila Research Conference, March 7-11 at the Sheraton Chicago Hotel & Towers. Specifically, researchers are trying to determine how, within organs, cells specialize while stem cells maintain tissues and enable them to repair damage and respond to stress or aging. Four talks, one on Thursday morning and three on Sunday morning, present variations on this theme.

For a fertilized egg to give rise to an organism made up of billions or trillions of cells, a precise program of cell divisions must unfold. Some divisions are "asymmetric": one of the two daughter cells specializes, yet the other retains the ability to divide. Chris Q. Doe, Ph.D., professor of biology at the University of Oregon, compares this asymmetric cell division to splitting a sundae so that only one half gets the cherry. The "cherries" in cells are the proteins and RNA molecules that make the two cells that descend from one cell different from each other. This collecting of different molecules in different regions of the initial cell before it divides is termed "cell polarity."

Dr. Doe and his team are tracing the cell divisions that form a fly's nervous system. "Producing the right cells at the right time is essential for normal development, yet it's not well understood how an embryonic precursor cell or stem cell generates a characteristic sequence of different cell types," he says. Dr. Doe and his team traced the cell lineages of 30 neuroblasts (stem cell-like neural precursors), each cell division generating a daughter cell bound for specialization as well as a self-renewing neuroblast. The dance of development is a matter of balance. Self-renew too much, and a tumor results; not enough, and the brain shrinks.

Tracing a cell lineage is a little like sketching a family tree of cousins who share a great-grandparent except that the great-grandparent (the neuroblast) continually produces more cousins. "The offspring will change due to the different environments they are born into," says Dr. Doe.

Julie A. Brill, Ph.D., a principal investigator at The Hospital for Sick Children (SickKids) in Toronto, investigates cell polarity in sperm cells. These highly specialized elongated cells begin as more spherical precursor cells. Groups of developing sperm elongate, align, condense their DNA into tight packages, expose enzyme-containing bumps on their tips that will burrow through an egg's outer layers, form moving tails, then detach and swim away.

The Brill lab studies a membrane lipid called PIP2 (phosphatidylinositol 4,5-bisphosphate) that establishes polarity in developing male germ cells in Drosophila. "Reducing levels of PIP2 leads to defects in cell polarity and failure to form mature, motile sperm," Dr. Brill says. These experiments show that localization of the enzyme responsible for PIP2 production in the growing end of elongating sperm tails likely sets up cell polarity. Since loss of this polarity is implicated in the origin and spread of cancer, defects in the regulation of PIP2 distribution may contribute to human cancer progression, she adds.

Stephen DiNardo, Ph.D., professor of cell and developmental biology at the Institute for Regenerative Medicine at the University of Pennsylvania, is investigating how different varieties of stem cells in the developing fly testis give rise to germ cells and epithelial cells that ensheathe the germ cells, as well as being able to self-renew. For each of these roles, stem cells are guided by their environment, known as their "niche."

In the fly testis, we know not only the locations of the two types of stem cells whose actions maintain fertility, but of neighboring cells. "We study how these niche cells are first specified during development, how they assemble, and what signals they use. Elements of what we and others learn about this niche may well apply to more complex niches in our tissues," Dr. DiNardo explains.

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Fly research gives insight into human stem cell development and cancer