Induced Pluripotent Stem Cells Market Demands, Trends, Growth … – MilTech

This report studies the global Induced Pluripotent Stem Cells market, analyzes and researches the Induced Pluripotent Stem Cells development status and forecast in United States, EU, Japan, China, India and Southeast Asia.

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This report was prepared as a study of the global market for induced pluripotent stem cells. Information is presented with a specific focus on the various market segments, including product-type-based, product-function-based, application-based, geography-based and technological-method-based. The detailed segmentation is to provide a deep profiling of the global iPSCs market based on which the market forecasts are made.

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Artificial Blood Vessels Mimic Rare Accelerated Aging Disease – Duke Today

Biomedical engineers have grown miniature human blood vessels that exhibit many of the symptoms and drug reactions associated with Hutchinson-Gilford Progeria Syndromean extremely rare genetic disease that causes symptoms resembling accelerated aging in children.

The technology will help doctors and researchers screen potential therapeutics for the disease more rapidly, with the goal of eventually creating a platform for personalized screening. The technique also offers a new way to study other rare diseases and could provide insights into treating heart disease in the elderly.

The study was published online on August 15 in the journal Scientific Reports.

"One of the drugs currently prescribed for this disease extends patients' lives by three months, and that's been considered a major feat," said Leigh Atchison, a doctoral candidate in biomedical engineering at Duke University and first author of the study. "They're looking for anything that will extend lifespan by even a few months. It's that devastating."

Hutchinson-Gilford Progeria Syndromeor simply progeria for shortis a non-hereditary genetic disease caused by a single-point mutation in the genome. It is so rare and so deadly that there are currently only about 250 known cases worldwide.

Progeria is triggered by a defective protein called progerin that accumulates outside of a cell's nucleus rather than becoming part of its structural support system. This causes the nucleus to take on an abnormal shape and inhibits its ability to divide. The resulting symptoms look much like accelerated aging, and affected patients usually die of heart disease brought on by weakened blood vessels before the age of 14.

"Progeria isn't considered hereditary, because nobody lives long enough to pass it on," said George Truskey, the R. Eugene and Susie E. Goodson Professor of Biomedical Engineering at Duke. "There are currently 75 children in clinical trials, which is amazing given the rarity of the disease. But with 15 compounds under consideration for trials, the math just ultimately won't work out. You can't try all of these drugs or various combinations of them in humans, so we're hoping our platform will provide an alternative way to test them."

Blood vessels are difficult to simulate because their walls have multiple layers of cells, including the endothelium and the media. The endothelium is the innermost lining of all blood vessels that interacts with circulating blood. The media is made mostly of smooth muscle cells that help control the flow and pressure of the blood within.

Researchers believe that it is the deterioration of these smooth muscle cells that ultimately leads to the heart disease and failure that so often kills patients with progeria. But because there are so few people with progeria, it is extremely difficult to study in the patients themselves.

"Because it's such a hard disease to study, we wanted to see if we could create a platform using human cells that more accurately represents the disease and then use it for drug testing," said Atchison. "So we tried to grow miniature artificial blood vessels using induced pluripotent stem cells derived from cells taken from patients with progeria."

The plan worked. In just four weeks of growth, the engineered blood vessels exhibit many of the symptoms seen in people with the diseasesymptoms that simple cell cultures have not been able to recreate. The blood vessels also respond similarly to pharmaceuticals, revealing nuances into how current therapies are working.

While the blood vessels showed improved function after a week of being dosed with an analogue of rapamycin, a drug known as everolimus, calcification and other symptoms of cardiovascular disease remained. This implies that the drug is helping the smooth muscle cells work better, but not remedying the underlying symptoms.

"That's why our system could be so useful," said Atchison. "It could tell us exactly what the drug is doing in a quicker, more high-throughput manner, and whether we need a second treatment to address other aspects of the disease."

The success may aid the study of other rare diseases, too.

"The major thing we're happy with is that this serves as a proof of principle for creating a vascular model of a rare disease in the laboratory to better understand it and hopefully develop a therapy," said Truskey.

The research may also provide insight into why some elderly people become especially prone to heart disease. Many heart patients have shown the same buildup of the progerin protein, so researchers believe there may be a link between the two conditions.

There are, of course, limitations to the new artificial blood vessels. They are not connected to any outside organs, nor are they embedded in the complicated biology of a living human being.

"We only created smooth muscle cells from progeria patients in this study, but their endothelial cells might play a major role as well," said Atchison. "If we can incorporate endothelial cells derived from the patients' own cells into the model as well, then we can create a more personalized testing platform for these patients."

This research was supported by the National Institutes of Health (UH3TR000505, R01HL126784), the National Science Foundation (GRFP Grant #1106401), the Maryland Stem Cell Research Fund, and the Progeria Research Foundation.

CITATION: "A Tissue Engineered Blood Vessel Model of Hutchinson-Gilford Progeria Syndrome Using Human iPSC-derived Smooth Muscle Cells," Leigh Atchison, Haoyue Zhang, Kan Cao, George A. Truskey. Scientific Reports, Aug. 15, 2017. DOI: 10.1038/s41598-017-08632-4

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Artificial Blood Vessels Mimic Rare Accelerated Aging Disease - Duke Today

Dopaminergic neurons derived from iPSCs in non-human primate model – Phys.Org

August 8, 2017 Stem Cells and Development is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. Credit: Mary Ann Liebert, Inc., publishers

Researchers have demonstrated the ability to generate dopaminergic neurons in the laboratory from induced pluripotent stem cells (iPSCs) derived from fibroblast cells of adult marmoset monkeys. This new study, documenting the iPSCs' pluripotent properties and the potential for using this animal model to develop regenerative medicine approaches for dopamine-related disorders such as Parkinson's disease, is published in Stem Cells and Development.

Marina Emborg, MD, PhD and colleagues from University of Wisconsin-Madison coauthored the article entitled "Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Adult Common Marmoset Fibroblasts." The researchers reported that the marmoset fibroblast-derived iPSCs could differentiate into all three embryonic cell lineages: mesoderm, ectoderm, and endoderm. When stimulated to pattern themselves as neurons, the iPSCs expressed genes and other biomarkers consistent with a dopaminergic phenotype.

"This important study advances the marmoset as a model for Parkinson's disease, for the first time deriving a line from the adult marmoset," says Editor-in-Chief Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

Explore further: Stem cell-derived dopaminergic neurons rescue motor defects in Parkinsonian monkeys

More information: Scott C. Vermilyea et al, Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Adult Common Marmoset Fibroblasts, Stem Cells and Development (2017). DOI: 10.1089/scd.2017.0069

Parkinson's disease is a degenerative disorder of the central nervous system that is characterized by tremors, rigidity, slowness of movement, and difficulty walking. It is caused by loss of the neurons that produce the neurotransmitter ...

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In the cells of palm trees, humans, and some single-celled microorganisms, DNA gets bent the same way. Now, by studying the 3-D structure of proteins bound to DNA in microbes called Archaea, University of Colorado Boulder ...

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How Food Preservatives May Disrupt Human Hormones – Laboratory Equipment

Can chemicals that are added to breakfast cereals and other everyday products make you obese? Growing evidence from animal experiments suggests the answer may be "yes." But confirming these findings in humans has faced formidable obstacles - until now.

A new study published today in Nature Communications details how Cedars-Sinai investigators developed a novel platform and protocol for testing the effects of chemicals known as endocrine disruptors on humans.

The three chemicals tested in this study are abundant in modern life. Butylhydroxytoluene (BHT) is an antioxidant commonly added to breakfast cereals and other foods to protect nutrients and keep fats from turning rancid; perfluorooctanoic acid (PFOA) is a polymer found in some cookware, carpeting and other products; and tributyltin (TBT) is a compound in paints that can make its way into water and accumulate in seafood.

The investigators used hormone-producing tissues grown from human stem cells to demonstrate how chronic exposure to these chemicals can interfere with signals sent from the digestive system to the brain that let people know when they are "full" during meals. When this signaling system breaks down, people often may continue eating, causing them to gain weight.

"We discovered that each of these chemicals damaged hormones that communicate between the gut and the brain," said Dhruv Sareen, PhD, assistant professor of Biomedical Sciences and director of the Induced Pluripotent Stem Cell Core Facility at the Cedars-Sinai Board of Governors Regenerative Medicine Institute. "When we tested the three together, the combined stress was more robust."

Of the three chemicals tested, BHT produced some of the strongest detrimental effects, Sareen said.

While other scientists have shown these compounds can disrupt hormone systems in laboratory animals, the new study is the first to use human pluripotent stem cells and tissues to document how the compounds may disrupt hormones that are critical to gut-to-brain signaling and preventing obesity in people, Sareen said.

"This is a landmark study that substantially improves our understanding of how endocrine disruptors may damage human hormonal systems and contribute to the obesity epidemic in the U.S.," said Clive Svendsen, PhD, director of the institute and the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine. More than one-third of U.S. adults are considered to be obese, according to federal statistics.

The new testing system developed for the study has the potential to provide a much-needed, safe and cost-effective method that can be used to evaluate the health effects of thousands of existing and new chemicals in the environment, the investigators say.

For their experiments, Sareen and his team first obtained blood samples from adults, and then, by introducing reprogramming genes, converted the cells into induced pluripotent stem cells. Then, using these stem cells, the investigators grew human epithelium tissue, which lines the gut, and neuronal tissues of the brain's hypothalamus region, which regulates appetite and metabolism.

The investigators then exposed the tissues to BHT, PFOA and TBT, one by one and also in combination, and observed what happened inside the cells. They found that the chemicals disrupted networks that prepare signaling hormones to maintain their structure and be transported out of the cells, thus making them ineffective. The chemicals also damaged mitochondria - cellular structures that convert food and oxygen into energy and drive the body's metabolism.

Because the chemical damage occurred in early-stage "young" cells, the findings suggest that a defective hormone system potentially could impact a pregnant mother as well as her fetus in the womb, Sareen said. While other scientists have found, in animal studies, that effects of endocrine disruptors can be passed down to future generations, this process has not been proved to occur in humans, he explained.

More than 80,000 chemicals are registered for use in the U.S. in everyday items such as foods, personal care products, household cleaners and lawn-care products, according to the National Toxicology Program of the U.S. Department of Health and Human Services. While the program states on its website that relatively few chemicals are thought to pose a significant risk to human health, it also states: "We do not know the effects of many of these chemicals on our health."

Cost and ethical issues, including the health risk of exposing human subjects to possibly harmful substances, are among the barriers to testing the safety of many chemicals. As a result, numerous widely used compounds remain unevaluated in humans for their health effects, especially to the hormone system.

"By testing these chemicals on actual human tissues in the lab, we potentially could make these evaluations easier to conduct and more cost-effective," Sareen said.

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ASU grad students’ lab skills help earn funding for cutting-edge biomedical research – Arizona State University

August 8, 2017

While clues to treating diseases that ravage the body and mind later in life sometimes appear in early stages of human development, studying a subjects entire lifespan is neither efficient nor practical.

So how can researchers study these early stages to combat diseases that manifest themselves later on? The stem cell wizards of ASUs Brafman Lab. Left to right: Sreedevi Raman, Josh Cutts, Nick Brookhouser and Christopher Potts. Photo by Marco-Alexis Chaira/ASU Download Full Image

If you guessed through the use of pluripotent stem cells so named because they have the ability to turn into other types of cells then youre right on the money.

ASUs Brafman Labis on the cutting edge of this branch of research, recently earning a $1.5 million grant from the National Institutes of Health to study the mechanisms of early human neurodevelopment, and $225,000 from the Arizona Biomedical Research Commission to study the effects of aging and other risk factors for Alzheimers Disease.

Biomedical engineering Assistant Professor David Brafman, who heads the laboratory, credits his graduate students as crucial in securing these grants

The graduate students hard work, creativity and dedication were critically important for generating the data to convince the reviewers that our approach was feasible and worth funding, Brafman says of his students. Too often the success of a lab is attributed to the [principal investigator] when it is the postdocs, grad students and research technicians who are down in the trenches doing the work.

Graduate students from the Brafman Lab reviewing data. Photo by Marco-Alexis Chaira/ASU

The students working in the Brafman Lab often labor late into the night and sometimes on the weekend. They possess a special mix of a passion for their work and the knowledge that achieving potentially life-altering outcomes dont come with a simple nine-to-five job.

The laboratory they work in combines developmental biology, genetic engineering and bioinformatics to investigate the various factors that can govern a stem cells fate. If they can figure out the mechanisms behind the stem cells multipotential futures, they could use that information to design targeted therapies for ailments like idiopathic pulmonary fibrosis, heart failure and Alzheimers Disease.

Take Josh Cutts, who is pursuing his doctoral degree in biomedical engineering. He knows a thing or three about working in the Brafman Lab. Hes addicted to the thrill of discovery, regardless of any challenges or obstacles that may come his way.

Were working on things that havent been done before so its challenging sometimes frustrating to complete certain experiments or understand the results, he said.

The shapeshifting nature of the stem cells can make working with them seem like biological wizardry. In the lab the research team has made stem cells into brain cells, heart cells, lung cells and more.

Now we are working with cutting-edge brain organoids known colloquially as mini brains, which sounds a little eerie, to address many different research questions, said Cutts, who earned his bachelors and masters degrees in biomedical engineering at California Polytechnic State University, San Luis Obispo. Its miraculous to work with these every day.

Cutts work generated the preliminary data that helped the lab secure the NIH grant. After finishing his graduate work, this pluripotent scholar plans to earn a post doctorate degree to expand his knowledge and expertise. Long term, he hopes to contribute to translating stem cell technology to patients, in academia or industry.

Researcher Nick Brookhouser is working toward his doctorate in clinical translational sciences at the University of Arizonas College of Medicine in Phoenix. His research in the Brafman Lab is focused on Alzheimers Disease and investigating the contribution of the Apolipoprotein E gene, or APOE, towards the diseases progression.

He has successfully generated a set of stem cell lines from Alzheimers patients as well as other stem cell lines that serve as the control group in his research. He is currently working with gene editing techniques to investigate APOEs relationship to Alzheimers.

Brookhousers work is also supported by an Arizona Biomedical Research Commission grant. He developed patient-specific pluripotent stem cell lines and brain cell lines, and with those lines he created a 3-D neural culture system that models a brain for study. He has also been involved in testing and optimizing gene editing technologies.

In the future, he hopes to transition to more clinical-based research in the biotechnology industry. Long term he hopes to contribute to the development of cell-based therapies and work in clinical trials.

Doctoral student Sreedevi Raman has also been working on research related to Alzheimers Disease. Instead of experimenting with stem cells at their genesis, Raman is trying to make them old. She is intentionally accelerating the aging process of cells in a dish so that they may be used to model various age-related disorders.

Her work with induced pluripotent stem cells specifically has helped the Brafman Lab attain the ABRC grant. Raman can take adult stem cells and program them back into state where their fate is not yet assigned.

Christopher Potts, a research specialist with a professional science masters degree from ASU, works with gene editing. His contribution to the team is comparable to using copy and paste for genes, but a bit more complicated. Hes using technologies like CRISPR (Clustered regularly interspaced short palindromic repeats) to edit stem cell genomes.

I am changing the DNA of stem cells. Thats pretty cool, right? Potts said. I think one of the coolest things about our lab is how each student has their own project and functions basically independently, but we all help each other and are able to do much more than we could on our own.

Hes enjoying his research, but also looks forward to teaching a new generation of students in the future. He has a masters degree in science education and taught high school for four years before joining the lab.

Potts has aspirations of starting a new line of scientists through a, career in outreach or other high-level science education.

The cells he works on use signaling pathways to regulate what they will become like his multiple career options. Right now, I am just hoping for some signals to help me differentiate, he said.

Just as Brafman relies on the hard work of his students, the entire lab team relies on one another to succeed.

Our lab is pretty close-knit. We like to hang out together to socialize and I think that support system makes our lab more effective, Cutts said. If any of us are having a hard time with experiments or anything at all, you can rely on your lab members and especially [Brafman] to help you work it out.

Like Cutts, Brookhouser values the highly collaborative environment in the lab that has fostered strong professional relationships as well as lasting friendships.

Just as patient somatic cells can be reprogrammed to a pluripotent state, I feel that the skills and mentorship I have gained in this lab have allowed me to reach a pluripotent state and primed me to differentiate down many different career paths in the future, Brookhouser said.

Raman credits her positive collaborative learning experiences in the lab with helping her to make advances in research as well as open career possibilities for her future. Since she just started her doctoral work, shes got a lot of research ahead of her. Luckily, she found a good place to start.

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ASU grad students' lab skills help earn funding for cutting-edge biomedical research - Arizona State University

CRISPR Corrects Disease Mutation in Human Embryos – Genetic Engineering & Biotechnology News (blog)

In an unexpected twist, the investigators discovered that once the paternal gene was excised, the genetic material originating from the mother (i.e., the homologous wild-type maternal gene) was more easily substituted than the synthetic DNA the scientists attempted to introduce.

To reduce mosaicism, which is characterized by a population of cells that originate from one egg but are genetically distinct, researchers injected sperm cells and CRISPR components directly into oocytes early in their cell-cycle phase, only 18 hours post-fertilization. The study authors assumed this would be the best time for genome editing to occur, as the sperm at that time only has a single mutant copy. In addition, injecting genetic material early, before DNA replication occurred, meant that the CRISPR components stayed in the cytoplasm longer. As a result of prolonged cytoplasm residency, the CRISPR components degraded quickly, before further replication of mutant alleles could occur.

Mosaicism, noted the authors, could have major negative effects and could restrict the clinical applications of the gene-editing technique in embryos, a fact that the authors identified as a limitation. In addition, the uncertainty surrounding the ability to reproduce the studys findings was also a limitation, the authors acknowledged.

Employing CRISPR in embryos, rather than in stem cells, yielded better results: The overall targeting efficiency in human embryos was found to be 72.2% (13/18), which was higher than the rate in induced pluripotent stem cells (iPSCs) exposed to the same construct (27.9%, or 17/61). The higher targeting efficiency suggests that human embryos employ different DNA repair mechanisms than do somatic or pluripotent cells, probably reflecting evolutionary requirements for stringent control over genome fidelity in the germline," the authors wrote in the paper.

Because off-target cutting is also a concern withCRISPR/Cas9, researchers evaluated all of the potential off-target sites via a whole-genome sequencing analysis. They determined that their technique did not produce any detectable off-target mutations in the blastomeres.

And, because Cas9 was used in purified protein form, and was not contained in a plasmid, off-site targeting was further reduced, investigators concluded.

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CRISPR Corrects Disease Mutation in Human Embryos - Genetic Engineering & Biotechnology News (blog)

World’s 1st trial of drug developed from iPS cells to begin – Japan … – Japan Today

Japanese researchers are set to begin the world's first clinical trial of a drug developed from iPS stem cells to treat a rare bone disease, Kyoto University said Tuesday.

A team led by Junya Toguchida, professor at the university, used so-called induced pluripotent stem cells to develop a potential drug for fibrodysplasia ossificans progressiva, or FOP, a rare disorder in which muscle tissue is gradually replaced by bone, inhibiting body movement.

The researchers created iPS cells from FOP patients and replicated the symptom outside their bodies. After adding components to the cells with features of the disease, they found an immune-suppressive agent called Rapamycin is effective for preventing abnormal bone formation.

The drug's safety and effectiveness need to be tested in a clinical trial, which could begin as early as September, on 20 patients aged 6 or older. A review committee at Kyoto University Hospital has already approved the trial.

The team has confirmed the effectiveness of Rapamycin in experiments with mice. Researchers gave the agent to mice after transplanting FOP patients' iPS stem cells into them and found out that the drug inhibited abnormal born formation.

"Rapamycin is a drug already used (for treatment of other diseases) so I expect patients will welcome" its use in the clinical trial, Toguchida said at a press conference.

Shinya Yamanaka, professor at Kyoto University and a 2012 Nobel Prize winner in medicine for discovering iPS cells, said, "I hope the clinical trial will spur active research for drug development and eventually lead to the discoveries of new treatment for various rare diseases."

The disease is caused by a mutation of a gene called ACVR1. Bones are formed in muscles, tendons and ligaments, hindering the movements of joints. Patients may experience difficulty in breathing if their respiratory muscles are affected.

iPS cells can grow into any type of human body tissue. They are expected to be utilized for drug development as well as regenerative medicine.

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World's 1st trial of drug developed from iPS cells to begin - Japan ... - Japan Today

Stem Cell Glossary – Closer Look at Stem Cells

Stem cell science involves many technical terms. This glossary covers many of the common terms you will encounter in reading about stem cells.

Adult stem cells A commonly used term for tissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

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Autologous Cells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

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Basic research Research designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

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Blastocyst A transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

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Bone marrow stromal cells A general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

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Cardiomyocytes The functional muscle cells of the heart that allow it to beat continuously and rhythmically.

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Clinical translation The process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

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Clinical trial Tests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

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Cord blood The blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

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Cytoplasm Fluid inside a cell, but outside the nucleus.

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Differentiation The process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

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Drug discovery The systematic process of discovering new drugs.

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Drug screening The process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

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Embryo Generally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

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Embryonic stem cells (ESCs) Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

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Fibroblast A common connective or support cell found within most tissues of the body.

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Glucose A simple sugar that cells use for energy.

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Hematopoietic Blood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

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Immunomodulatory The ability to modify the immune system or an immune response.

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Induced pluripotent stem cells (iPSCs) Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

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In vitro Latin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

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In vivo Latin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

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Islets of Langerhans Clusters in the pancreas where insulin-producing beta cells live.

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Macula A small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

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Mesenchymal stem cells (MSCs) A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

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Multipotent stem cells Stem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

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Neuron An electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

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Pancreatic beta cells Cells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

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Photoreceptors Rod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

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Placebo A pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

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Placebo effect Perceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

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Pluripotent stem cells Stem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

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Preclinical research Laboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

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Precursor cells An intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

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Progenitor cells An intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

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Regenerative Medicine An interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

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Reprogramming In the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

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Retinal pigment epithelium A single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

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RNA Ribonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

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Scientific method A systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

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Self-renewal A special type of cell division in stem cells by which they make copies of themselves.

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Somatic stem cells Scientific term for tissue-specific or adult stem cells.

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Stem cells Cells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

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Stem cell tourism The travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

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Teratoma A benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

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Tissue A group of cells with a similar function or embryological origin. Tissues organize further to become organs.

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Tissue-specific stem cells Stem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

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Totipotent The ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

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Translational research Research that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

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Zygote The single cell formed when a sperm cell fuses with an egg cell.

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Stem Cell Glossary - Closer Look at Stem Cells

What are induced pluripotent stem cells or iPS cells? – Stem …

In November 2007 scientists announced they had developed a new way to cause mature human cells to resemble pluripotent stem cells - similar in many ways to human embryonic stem cells. By simply altering the expression of just four genes using genetic modification, the mature cells were 'induced' to become more primitive, stem cells and were referred to as 'induced' pluripotent stem (iPS) cells.

Initially iPS cells were generated using viruses to change gene expression, however since the initial discovery, technologies for reprogramming cells are moving very quickly and researchers are now investigating the use of new methods that do not use viruses which can cause permanent and potentially harmful changes in the cells. If they are able to be made safely, and on a large scale, iPS cells could possibly be used to provide a source of cells to replace cells damaged following illness or disease. It may even be possible to make stem cells for therapy from a patient's own cells and thereby avoid the use of anti-rejection medications.

However, right now scientists are using this method to create disease specific cells for research by taking a cells - maybe from a skin biopsy - from a patient with a genetic disorder, such as Huntingtons disease, and then using the iPS cells to study the disease in the laboratory. Scientist hope that such an approach will help them understand the development and progression of certain diseases, and assist in the development and testing of new drugs to treat disease.

While the discovery of iPS cells was a very important development, more research needs to be done to discover if they will offer the same research value as embryonic stem cells and if they will be as useful for therapy.

To learn more about iPS cells watch What are induced pluripotent stem cells? in our video library.

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What are induced pluripotent stem cells or iPS cells? - Stem ...

A New Epigenetic Barrier to Induced Pluripotent Stem Cells – WhatIsEpigenetics.com

By adding theright concoction of ingredients, scientists can reprogram youreverydaysomatic cell intoan inducedpluripotentstemcell(IPSC) that is,aculturedcellthat has the ability todifferentiate into almost any othercelltypein response to specificenvironmentalfactors, similar to an embryonic stem cell. Thisinnovativetechnology allows the study of the molecularmechanismsofearlydevelopmentanddisease,withouttheethical restrictionsassociated withembryonic stem cells.

Not surprisingly, the possibility of utilizing induced pluripotent stem cells in the field of regenerative medicine is of important focus to many scientists. In a recent post, we touched on the potential ability of vitamins A and C to enhance the erasure of epigenetic memory required for cell reprogramming. Because these special types of cells can propagate indefinitely and form any other cell type in the body such as neurons, liver, and heart cells we may be able to replace lost organs, repair tissue, and even generate type O red blood cells, which can be used in transfusions for people with any blood type.

Greatso whats the problem?

Unfortunately, thereare drawbacksto this technology, namely the efficiency of reprogramming.Many IPSCsdo not actually gain completepluripotency. Epigenetic modifications are heavily implicated during the reprogramming process whereby the epigenetic makeup of the cell is completely overhauledto first encourage the expression of pluripotent genes and thenremodelled to encourage the expression of genes associated withthefinalcell typethattheIPSCwillbecome. As the epigenome plays a crucial role inreprogramming,inconsistenciesof pluripotencyacrossIPSClinesmaybedue toepigenetic barriers.

TRIM28: a novel epigenetic barrier

A team of scientists headed by Dr. Miles from The Netherlands Cancer institute recently uncovered a novel epigenetic barrier to the efficient induction of pluripotent stem cell reprogramming. Published in a recent issue of STEM CELLS, the paper highlights the use of a shRNA screen targeting over 670 epigenetic modifiers, revealing the involvement of TRIM28 in the resistance of cells transitioning from somatic to pluripotent state.

TRIM28, or Tripartite motif-containing 28, is involved in mediating transcriptional control by interacting with a certain domain in numerous transcription factors. Previous research shows that it plays a role in cellular differentiation and proliferation, DNA damage repair response, transcriptional regulation, and apoptosis.

By blocking the expression TRIM28 during reprogramming, the group demonstrated increases in the number of cells reaching pluripotency, as well as increased expression of a selection of 143 genes.

Analysis of the list of genes revealed the most statistically significant gene ontology term was unclassified. This result indicates TRIM28 does not regulate a specific pathway during reprogramming, states the authors.

It is known that TRIM28 gene encodes for a protein known to be involved in transcriptional regulation via the recruitment and formation of protein complexes that maintain repressive chromatin. Given this, researchers proposed the gene expression alterations, hence reprogramming differences, were likely to be associated with chromatin modification.

SEE ALSO: Maternal Smoking Epigenetically Harms Child Development

Subsequent tests supported this notion by establishing a proportion of the 143 genes to be located near H3K9me3 a repressive histone H3 modification which has shown to influence the transcription of genes that impedes the IPSC reprogramming process. When TRIM28 expression was blocked, the closer genes are to the H3K9me3 the greater the increase in expression. This suggests the role of TRIM28 in repressing the expression of genes involved in reprogramming via the maintenance of H3K9me3 heterochromatin site.

Whyis this important?

Due to the potential to produce almost anyothertype of cell, thetechnology ofIPSChas sparked excitement in the clinical sciences. The implementation ofIPSCto repair damaged or diseased tissue or to test/develop personalised medicines ison the horizon.By establishing barriers preventing the efficient transition of differentiated cells to pluripotent cells scientist canrefineIPSC generationto make the future clinical use ofIPSCsboth safe and efficient.

Source: Miles, D. C., de Vries, N. A., Gisler, S., Lieftink, C., Akhtar, W., Gogola, E., & Beijersbergen, R. L. (2017). TRIM28 is an Epigenetic Barrier to Induced Pluripotent Stem Cell Reprogramming.STEM CELLS,35(1), 147-157.

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A New Epigenetic Barrier to Induced Pluripotent Stem Cells - WhatIsEpigenetics.com