$2.4 million toward gene therapy for human degenerative retinal diseases (12/5/2008)

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. 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.

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.

Dr. Robert Koenekoop 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 McGill University. His research is also funded by the Foundation Fighting Blindness Canada and the Fonds de la recherche en santé du Québec (FRSQ).

Note: This story has been adapted from a news release issued by the McGill University Health Centre

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