Category Archives: Regenerative Medicine

WSCS 2014: DEVELOPMENT OF JAPANESE REGENERATIVE MEDICINE INDUSTRY
Presenter - Takuya Yokokawa, FujiFilm.

By: worldstemcell

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WSCS 2014: DEVELOPMENT OF JAPANESE REGENERATIVE MEDICINE INDUSTRY - Video

Cormac Murphy went straight from an undergraduate degree in zoology at Trinity College Dublin to a taught masters degree (MSc) in regenerative medicine at NUI Galway.

Originally from Drumcondra in Dublin, the 23 year-old moved to Galway for the one-year full-time course.

After doing his undergraduate thesis in developmental biology, he decided to move to a health-related science. His supervisor told him about the Regenerative Medicine Institute and NUI Galway and thought he might enjoy it.

It had a really good reputation, and she thought it would be a good springboard to further things, Murphy said. In undergrad I really enjoyed science and lab time and stuff like that, but I wasnt quite sure whether I wanted to go on to do a full PhD.

Its the only regenerative medicine masters degree in Ireland and one of the few in Europe. The course is a combination of lectures, lab time and continuous assessment.

Its about getting research from the lab to the clinic, which is what Im interested in and focused on: making clinical products that will actually help people.

Murphy says hes learning a lot about stem cell biology, and the course includes things like immunology and pharmacology. He has taken an elective business course. Thats something we scientists dont tend to know a lot about, but its important.

Over the summer, students will do independent research projects in the labs. Murphys project involves taking skin cells and attempting to turn them into the photoreceptors at the back of the retina: rods and cones.

It sounds kind of like magic. Thats why I was interested in it, he said.

Murphy hopes to move onto a PhD in the regenerative area after the masters, but he might take a year out first to work in industry. His long-terms plan is research and possibly lecturing.

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Postgrad profile: Cormac Murphy (MSc in regenerative medicine)

Cash reserves at the end of December fell to $149.2 million, from $196.4 million at the end of June. Photo: Erin Jonasson

Regenerative medicine group Mesoblast has refused to rule out raising fresh funds from the sharemarket as it continues to burn about $25 million a quarter in its stem cell research programs, which has left it with an "18-month runway" with its existing cash reserves.

However, the preferred option is to pursue partnerships with other drug companies.

"There are a number of strategic partnerships" covering its tier one and tier two candidate treatments, chief financial officer Paul Hodgkinson said.

"Our disc program is our most advanced unpartnered program," chief executive Silviu Itescu said. "A partner with an established sales force would be the appropriate partner for us ... and they would take on the full cost of our development program."

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It was the same with the company's rheumatoid arthritis research program, Mr Itescu said.

"Discussions are active and ongoing ... and clinical developments will be taken by our partner and the expected upfront [payment] would add substantially to our runway."

He also pointed to the prospects for attracting funds from Japan, Europe and the United States for the company's research.

Cash reserves at the end of December fell to $149.2 million, from $196.4 million at the end of June.

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Mesoblast refuses to rule out share raising

(SACRAMENTO, Calif.) - The California Institute for Regenerative Medicine (CIRM) has awarded a pair of $1.8 million grants to two UC Davis scientists to develop better tools for enabling physicians to assess the safety and efficacy of bioengineered tissues used to treat cardiovascular disease and bone and cartilage repair. Laura Marcu, a professor of biomedical engineering and neurological surgery, and her colleagues, aim to combine label-free optical and ultrasonic imaging technologies so that newly developed vascular replacement materials - typically used in surgical grafts to restore blood flow - can be better assessed and monitored directly in patients and thus prevent a graft's potential failure. Kent Leach, an associate professor of biomedical engineering and orthopaedic surgery, will lead an interdisciplinary research team that will also develop advanced light and sound technologies to detect changes in engineered bone and cartilage. The goal of the project is to ultimately provide clinicians with improved abilities to identify if and how implanted cells are maturing and functioning in patients. Having these types of new diagnostic imaging capabilities could accelerate the development and clinical applications of everything from engineered vascular grafts, which currently can pose significant complications for patients, to stem cell therapies for regenerating bone and cartilage in diseased or damaged areas of the human body. "The broad range of biophotonic and ultrasound technologies developed in our laboratory could improve our ability to produce safer, more functional engineered tissues in the laboratory and large animal models to speed their use in clinical settings," said Marcu, who also is co-principal investigator on Leach's research project. "It should improve real-time, non-invasive, label-free imaging capabilities and give us a more thorough assessment of site-specific cellular growth and functional properties when engineered tissues are used." The two CIRM grants are part of the state stem cell agency's latest Tools and Technologies Initiative, which is designed to support research that addresses unique translational challenges in regenerative medicine. The three-year research grants that were awarded by the agency last week focus on the creation, design and testing of novel or existing tools and technologies to address translational bottlenecks to stem cell therapies. "Sometimes even the most promising therapy can be derailed by a tiny problem," said Jonathan Thomas, chair of the CIRM Board, in a statement regarding the nearly $30 million in grants that stem cell agency approved at its Jan. 29 meeting. "These awards are designed to help find ways to overcome those problems, to bridge the gaps in our knowledge and ensure that the best research is able to keep progressing and move out of the lab and into clinical trials in patients." In her CIRM proposal, Marcu noted that cardiovascular disease, when combined with rising rates of peripheral artery disease and ischemic stroke, make the illness the most prominent health problem in California and the United States. "Our goal is to help develop a diagnostic technology that is more practical and less costly than what is currently available," said Marcu, whose project is being done in collaboration with Leigh Griffiths at the UC Davis School of Veterinary Medicine and Claus Sondergaard at the UC Davis School of Medicine. "We want to be able to more rapidly screen vascular scaffold production and real-time assessments, on an ongoing basis, of bioengineered vascular tissues after studies are performed in patients or in animals. Having that type of ability could allow clinicians to more readily identify early signs of rejection and vascular graft failure and thereby improve safety and efficacy for use in patients." As new therapies are developed to treat the disease, especially involving tissue grafts and patches engineered with unique cellular material, the need for devices to test and monitor bioengineered products will be all the more important. "Currently, using stem cells to generate individualized implantable grafts suffers from patient-to-patient variability that is unpredictable and immeasurable without destructive techniques," said Leach, whose project also includes Kyriacos Athanasiou, chair of UC Davis' Department of Biomedical Engineering. "The aging population of California, 20 percent of whom will be over the age of 65 in the next decade, will require functional replacement tissues to maintain their quality of life. We simply cannot assess the success or failure of a cellular therapy in living individuals, and it represents a major bottleneck in translating stem cell technologies to the clinic and delivering quality products for patients. We need nondestructive, minimally invasive methods to measure dynamic changes in tissue development." UC Davis' stem cell program director, Jan Nolta, also sees great benefits for regenerative medicine research from the tools Marcu and Leach are developing. "One of the great barriers in regenerative medicine is our ability to understand and monitor what happens after stem cells are given to a patient," said Nolta, who also directs the UC Davis Institute for Regenerative Cures in Sacramento. "We need to be able to tell whether the blood vessels are truly improving, and whether the bone and cartilage are getting stronger. This type of novel biomedical imaging research will advance our clinical assessment capabilities and add to our efforts to safely turn stem cells into cures."

For more information, visit http://www.ucdmc.ucdavis.edu/stemcellresearch

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$3.6 Million in Grants for Pair of UC Davis Scientists

P-SPAN #411: "Treating Blindness with Stem Cells"
On February 5, 2015, the Science/Biotechnology Department at Berkeley City College kicked off their Spring 2015 Seminar Series, sponsored by the California Institute for Regenerative Medicine....

By: Peralta Colleges

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P-SPAN #411: "Treating Blindness with Stem Cells" - Video

WSCS 2014: REGENERATIVE MEDICINE: A NEW ERA OF DISCOVERY AND INNOVATION
Moderator - John Sterling, Genetic Engineering Biotechnology News Speakers - Marie Csete, MD, PhD, Huntington Medical Research Institute Aubrey de Grey, ...

By: worldstemcell

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WSCS 2014: REGENERATIVE MEDICINE: A NEW ERA OF DISCOVERY AND INNOVATION - Video

WSCS 2014: REGENERATIVE MEDICINE FOR AGING: MAKING REJUVENATION COMPREHENSIVE NOT COSMETIC
Presenter - Aubrey de Grey, PhD, SENS Foundation.

By: worldstemcell

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WSCS 2014: REGENERATIVE MEDICINE FOR AGING: MAKING REJUVENATION COMPREHENSIVE NOT COSMETIC - Video

Stem Cell Sound Bites: Duchenne Muscular Dystrophy
Visit: http://www.uctv.tv/) Carrie Miceli and Stanley Nelson of UCLA describe their efforts to use stem cell-based strategies to find a drug treatment for d...

By: University of California Television (UCTV)

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Stem Cell Sound Bites: Duchenne Muscular Dystrophy - Video

US MILITARY INVESTMENT IN REGENERATIVE MEDICINE HD
Moderator - Michael R. Davis, MD, FACS, United States Army Institute of Surgical Research Speakers - Debra Niemeyer, PhD, 59th Medical Wing Joint Base San An...

By: worldstemcell

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US MILITARY INVESTMENT IN REGENERATIVE MEDICINE HD - Video

When it comes to gene expression -- the process by which our DNA provides the recipe used to direct the synthesis of proteins and other molecules that we need for development and survival -- scientists have so far studied one single gene at a time. A new approach developed by Harvard geneticist George Church, Ph.D., can help uncover how tandem gene circuits dictate life processes, such as the healthy development of tissue or the triggering of a particular disease, and can also be used for directing precision stem cell differentiation for regenerative medicine and growing organ transplants.

The findings, reported by Church and his team of researchers at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School in Nature Methods, show promise that precision gene therapies could be developed to prevent and treat disease on a highly customizable, personalized level, which is crucial given the fact that diseases develop among diverse pathways among genetically-varied individuals. Wyss Core Faculty member Jim Collins, Ph.D., was also a co-author on the paper. Collins is also the Henri Termeer Professor of Medical Engineering & Science and Professor in the Department of Biological Engineering at the Massachusetts Institute of Technology.

The approach leverages the Cas9 protein, which has already been employed as a Swiss Army knife for genome engineering, in a novel way. The Cas9 protein can be programmed to bind and cleave any desired section of DNA -- but now Church's new approach activates the genes Cas9 binds to rather than cleaving them, triggering them to activate transcription to express or repress desired genetic traits. And by engineering the Cas9 to be fused to a triple-pronged transcription factor, Church and his team can robustly manipulate single or multiple genes to control gene expression.

"In terms of genetic engineering, the more knobs you can twist to exert control over the expression of genetic traits, the better," said Church, a Wyss Core Faculty member who is also Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and MIT. "This new work represents a major, entirely new class of knobs that we could use to control multiple genes and therefore influence whether or not specific genetics traits are expressed and to what extent -- we could essentially dial gene expression up or down with great precision."

Such a capability could lead to gene therapies that would mitigate age-related degeneration and the onset of disease; in the study, Church and his team demonstrated the ability to manipulate gene expression in yeast, flies, mouse and human cell cultures.

"We envision using this approach to investigate and create comprehensive libraries that document which gene circuits control a wide range of gene expression," said one of the study's lead authors Alejandro Chavez, Ph.D., Postdoctoral Fellow at the Wyss Institute. Jonathan Schieman, Ph.D, of the Wyss Institute and Harvard Medical School, and Suhani Vora, of the Wyss Institute, Massachusetts Institute of Technology, and Harvard Medical School, are also lead co-authors on the study.

The new Cas9 approach could also potentially target and activate sections of the genome made up of genes that are not directly responsible for transcription, and which previously were poorly understood. These sections, which comprise up to 90% of the genome in humans, have previously been considered to be useless DNA "dark matter" by geneticists. In contrast to translated DNA, which contains recipes of genetic information used to express traits, this DNA dark matter contains transcribed genes which act in mysterious ways, with several of these genes often having influence in tandem.

But now, that DNA dark matter could be accessed using Cas9, allowing scientists to document which non-translated genes can be activated in tandem to influence gene expression. Furthermore, these non-translated genes could also be turned into a docking station of sorts. By using Cas9 to target and bind gene circuits to these sections, scientists could introduce synthetic loops of genes to a genome, therefore triggering entirely new or altered gene expressions.

The ability to manipulate multiple genes in tandem so precisely also has big implications for advancing stem cell engineering for development of transplant organs and regenerative therapies.

"In order to grow organs from stem cells, our understanding of developmental biology needs to increase rapidly," said Church. "This multivariate approach allows us to quickly churn through and analyze large numbers of gene combinations to identify developmental pathways much faster than has been previously capable."

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Activating genes on demand: Possible?