QUEBEC, Feb. 4, 2013 /CNW Telbec/ - In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene
Therapy of Quebec and researcher in the Research center of the Centre
Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50
other world experts in gene therapy - for the creation of an
International Gene Therapy Consortium for Monogenic Diseases. The bases
of this consortium will be established during a workshop, which will be
held during the congress of the American Society of Gene and Cell
Therapy (ASGCT) in Salt Lake City in May 2013.
Recent scientific progress in molecular biology and in genomics allowed
during the recent years to identify the genes responsible for 10,000
hereditary diseases caused by a mutation in a single gene (for ex.,
Duchenne muscular dystrophy, Friedreich ataxia, haemophilia, etc.). On
the other hand, recent progress of gene therapy resulted in treatments
for some of these diseases previously considered incurable: hereditary
immuno-deficiencies (the bubble children), a form of hereditary
blindness (congenital amaurosis of Leber), etc. Also, for the first
time, a gene-therapy treatment was approved for commercialization in
Europe (for familial hyperchylomicronemia, a lipid disease). A Quebec
team participated in the development of this treatment. The discovery
of pluripotent stem cells, for which Dr. Yamanaka obtained the Nobel
Prize in Medicine 2012, also allows to genetically correct the patient
own cells and to differentiate them in various types of cells including
those of heart and brain. These cells could then be re-transplanted to
the patient without immunosuppression.
The research to develop treatments for these hereditary illnesses is at
present made by small teams often financially supported by small
patient associations. This fragmentation of the research and the
sub-financing make more difficult the development of clinical trials.
Professor Tremblay and his cosignatories indicate that with sufficient
budgets, it would be possible to develop globally therapies for the
most of these diseases during the next 2 decades.
The Regroupement québécois des maladies orphelines (Quebec Coalition of
Orphan Diseases) encourages initiatives that help develop treatments
for rare genetic disorders.
The article that calls for the creation of the Consortium :
Translating the Genomics Revolution: The Need for an International Gene
Therapy Consortium for Monogenic Diseases, Molecular Therapy, February 2013.
SOURCE: Regroupement québécois des maladies orphelines
For further information:
Jacques P. Tremblay
Professor, Department of Molecular Medicine
Université Laval, Québec.
418-654-2186 ou 418-262-6559
Gail Ouellette, Ph.D., CGAC
Director, Quebec Coalition of Orphan Diseases