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 qubcois 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 qubcois des maladies orphelines

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Creation of an International Gene Therapy Consortium