Harvard Stem Cell Institute researcher George Q. Daley, MD, PhD, also associate director of the Stem Cell Program at Children’s Hospital Boston, and HSCI colleagues Konrad Hochedlinger and Chad Cowan have produced a robust new collection of disease-specific stem cell lines, all of which were developed using the new induced pluripotent stem cell (iPS) technique. The paper is being published in the August 6 on-line edition of the journal Cell.
The new iPS lines, developed from the cells of patients ranging in age from one month to 57-years-old and suffering from a range of conditions from Down Syndrome to Parkinson’s disease, will be deposited in a new HSCI “core” facility being established at Massachusetts General Hospital (MGH), HSCI co-director Doug Melton announced yesterday. The operations of the iPS Core will be overseen by a faculty committee, which Daley will chair.
The cell lines the researchers produced carry the genes or genetic components for 10 different diseases, including Parkinson’s Disease, Type I diabetes, Huntington’s Disease, Down Syndrome, a form of combined immunodeficiency (“Bubble Boy’s Disease”), Lesch-Nyhan syndrome, Gaucher’s Disease, and two forms of Muscular Dystrophy, among others.
“We wanted to produce a large number of disease models for ourselves, our collaborators, and the stem cell research community to accelerate research,” Daley said. “The original embryonic stem cell lines are generic, and allow you to ask only basic questions. But these new lines are valuable tools for attacking the root causes of disease. Our work is just the beginning for studying thousands of diseases in a petri dish,” he said.
Melton said that the HSCI iPS Core will serve as a repository for iPS cells produced by HSCI scientists. “The Core will also function as a technical laboratory to produce these disease- specific lines for use by scientists around the world,” Melton said. He went on to say that “the suite of iPS cell lines reported by the Daley group marks an important achievement and a very significant advance for patients suffering from degenerative diseases.
These disease-specific iPS cells are invaluable tools that will allow researchers to watch the development diseases in petri dishes, outside of the patients. And we have good reason to believe that this will make it possible to find new treatments, and eventually drugs, to slow or even stop the course of a number of diseases. In years ahead,” Melton said, “this report will be seen as opening the door to a new approach to develop therapies."
"One of our goals in creating the NIH Director's Pioneer Award programs was to enable exceptionally creative scientists to move quickly in promising new directions, thereby speeding the intellectual and technical breakthroughs needed to address major challenges in biomedical or behavioral research," said National Institutes of Health Director Elias A. Zerhouni, M.D. "This is certainly the case for Drs. Daley and Hochedlinger, who deployed their Director's award resources to advance our ability to use induced pluripotent stem cells for disease-specific studies and drug development."
Daley and his colleagues, led by first-author and Children’s researcher In Hyun Park, PhD, intentionally produced some stem cell lines for highly heritable, single-gene diseases, such as Gaucher’s; complex genetic syndromes, such as Down; and then complex diseases, such as Parkinson’s, that involve genetic, cellular, and perhaps environmental components.
“The cell lines available from the iPS Core will allow stem cell researchers around the world to explore possible gene therapies for some conditions, and will aid in the development of drugs for others,” Daley said.
While Daley, President of the International Society for Stem Cell Research, is enthusiastic about the promise of reprogramming studies, he is far from ready to abandon experiments with embryonic stem cells. Daley believes that reprogramming and ESC research must advance in tandem to bring cell therapy to the clinic as quickly as possible.
The study was supported by grants from the National Institutes of Health (NIH), an NIH Director's Pioneer Award of the NIH Roadmap for Medical Research, an NIH Innovator’s Award, the Burroughs Wellcome Fund, the Leukemia and Lymhoma Society, the Harvard Stem Cell Institute, Children’s Hospital Boston Stem Cell Program, the Stowers Medical Institute, and the Howard Hughes Medical Institute.
22.02.2018 | Albert-Ludwigs-Universität Freiburg im Breisgau
Separate brain systems cooperate during learning, study finds
22.02.2018 | Brown University
Quantum computers may one day solve algorithmic problems which even the biggest supercomputers today can’t manage. But how do you test a quantum computer to...
For the first time, a team of researchers at the Max-Planck Institute (MPI) for Polymer Research in Mainz, Germany, has succeeded in making an integrated circuit (IC) from just a monolayer of a semiconducting polymer via a bottom-up, self-assembly approach.
In the self-assembly process, the semiconducting polymer arranges itself into an ordered monolayer in a transistor. The transistors are binary switches used...
Breakthrough provides a new concept of the design of molecular motors, sensors and electricity generators at nanoscale
Researchers from the Institute of Organic Chemistry and Biochemistry of the CAS (IOCB Prague), Institute of Physics of the CAS (IP CAS) and Palacký University...
For photographers and scientists, lenses are lifesavers. They reflect and refract light, making possible the imaging systems that drive discovery through the microscope and preserve history through cameras.
But today's glass-based lenses are bulky and resist miniaturization. Next-generation technologies, such as ultrathin cameras or tiny microscopes, require...
Scientists from the University of Zurich have succeeded for the first time in tracking individual stem cells and their neuronal progeny over months within the intact adult brain. This study sheds light on how new neurons are produced throughout life.
The generation of new nerve cells was once thought to taper off at the end of embryonic development. However, recent research has shown that the adult brain...
15.02.2018 | Event News
13.02.2018 | Event News
12.02.2018 | Event News
22.02.2018 | Life Sciences
22.02.2018 | Physics and Astronomy
22.02.2018 | Earth Sciences