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CA$2.4 million toward gene therapy for human degenerative retinal diseases
Date:12/3/2008

This release is available in French.

Montreal, December 3rd, 2008 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
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Contact: Isabelle Kling
isabelle.kling@muhc.mcgill.ca
514-843-1560
McGill University Health Centre
Source:Eurekalert

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