$2.4 million towards gene therapy for human degenerative retinal diseases
A Canadian and American research group including the team of Dr.
Robert Koenekoop from the Research Institute at the Montreal
Children's Hospital of the MUHC has just been awarded $2.4 million
from the Canadian Institutes of Health Research (CIHR) and the
Foundation Fighting Blindness Canada (FFB). This five-year grant
will fund an ambitious research project to develop innovative gene
therapies for a number of human degenerative retinal
diseases.
A multidisciplinary and complementary team
The complementary skills of the five research teams involved will
provide the multidisciplinarity required for success in this
research project. The project will be led by Dr. Robert Molday, a
cell biologist from the University of British Columbia. The team's
other experts in gene therapy are Dr. Jim Hu from the University of
Toronto, and Dr. Bill Hauswirth from the University of Florida. Dr.
Marinko Sarunic of Simon Fraser University will be responsible for
the retinal imaging component of the project.
As the team's clinician-scientist, Dr. Robert Koenekoop will
oversee the visual function testing and the gene analyses, first in
animals and then in humans with a variety of retinal degenerations.
After several years of testing in animals, human patients will be
injected with the "new healthy gene" that aims to partially restore
vision.
Innovative gene therapies
"We hope to begin a human gene-therapy trial in Canada within five
years for three specific degenerative retinal diseases: Leber
Congenital Amaurosis (LCA), Stargardt macular dystrophy and
retinitis pigmentosa," Dr. Koenekoop explained. "Previous attempts
with the gene named RPE65 have been highly conclusive for LCA: we
believe that we can learn from that and advance even more quickly
this time. It's very motivating!"
RPE65 mutations are one cause of Leber Congenital Amaurosis. Three
independent research teams have very recently shown that injecting
a healthy version of that gene to young adults can partially
restore their vision.
The CIHR's Regenerative Medicine and Nanomedicine
Initiative
Nine projects were awarded grants through the CIHR program entitled
"Regenerative Medicine and Nanomedicine - Emerging Team Grants -
July 2008." All of these projects hold great hope for medical
applications in the fields of nanotechnology, stem cells, tissue
engineering and rehabilitative sciences.
To receive the grant, the team had to show a multidisciplinary
commitment to addressing problems in regenerative medicine. The
long-term goal of this program is to develop innovative treatments
that are scientifically based and socially validated.
is the Director of the Division
of Pediatric Ophthalmology and the McGill Ocular Genetics
Laboratory at the Montreal Children's Hospital of the MUHC. He is a
researcher in medical genetics and genomics with the Research
Institute at the Montreal Children's Hospital of the MUHC. Dr.
Koenekoop is an Associate Professor in Human Genetics and
Ophthalmology in the Faculty of Medicine at Ï㽶ÊÓƵ. His
research is also funded by the Foundation Fighting Blindness Canada
and the Fonds de la recherche en santé du Québec (FRSQ).
The Research Institute of the Ï㽶ÊÓƵ Health
Centre (RI MUHC) is a world-renowned biomedical and
health-care hospital research centre. Located in Montreal, Quebec,
the institute is the research arm of the MUHC, the university
health center affiliated with the Faculty of Medicine at McGill
University. The institute supports over 600 researchers, nearly
1200 graduate and post-doctoral students and operates more than 300
laboratories devoted to a broad spectrum of fundamental and
clinical research. The Research Institute operates at the forefront
of knowledge, innovation and technology and is inextricably linked
to the clinical programs of the MUHC, ensuring that patients
benefit directly from the latest research-based knowledge.
The Research Institute of the MUHC is supported in part by the
Fonds de la recherche en santé du Québec.
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