Daley and colleagues create 20 disease-specific stem cell lines
Lines to be part of new HSCI iPS collection available to researchers
Boston, Mass, Aug. 7, 2008 – 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.
For a copy of the paper, please contact cgenova@cell.
CELL-D-08-00688R2, Daley
Public release date: 7-Aug-2008
Contact: Bess Andrews
Elizabeth.Andrews@
617-919-3103
Children's Hospital Boston
http://www.eurekale
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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