The AFM is continuing its support for projects aimed at overcoming the obstacles so far identified : improving vectors, ensuring their distribution throughout the body, producing them in conditions adequate for clinical trials etc.
In this way it has been contributing to the funding of recognised centres of excellence for several years. In particular: the Nantes centre of infrastructures, composed of a vectorology laboratory, a cell and gene therapy unit, a platform for the study of muscle transcripts and a centre for preclinical experimentation on models of rare diseases; the University Medical Centre of Geneva – a reference centre for the lentivirus, and the Gene Therapy Centre in Barcelona.
Both at the methodological as well as financial levels, the AFM is supporting the step to clinical experimentation for Leber amaurosis (F. Rolling, Nantes), Hurler and Sanfilippo diseases (JM. Heard ; O. Danos, Paris) Steinert myopathy (J. Puymirat, Quebec) and Duchenne myopathy (Transgène, Strasbourg ; Prosensa, Netherlands).
Research into vectorology and immunology is also carried out at Généthon, the laboratory of the AFM dedicated to the development of gene and cell therapies since 1997. Généthon is also exploring innovative leads in gene therapy transfer or gene surgery.
To know more
Gene therapy means integrating a healthy gene in the genome to compensate for the dysfunction of a defective gene in a cell. The main problem today involves vectors. Although the strategy of gene transfer is accepted today in principle,scientists are working on improving the tools used for gene therapy.
With genetic diseases, gene therapy involves replacing the function of a defective or missing gene with that of a therapeutic gene. Researchers have developed several types of vectors and techniques for gene transfer: viral vectors (a virus whose pathogenic elements have been replaced by the gene-drug), synthetic vectors (chemical compounds that bind to the therapeutic gene) and « physical » techniques (electroporation, biolistics, ...). Each method has its own advantages and disadvantages. One of the unique features of Généthon is that it works on all these vectors in order to determine the value of each and to use them for the suitable diseases.
The novel nature of this therapy should be underlined. All innovative therapies require a period of experimentation during which failures may occur, of which scientists should take advantage to advance further.
Généthon (In English)
Created in 1990 by AFM with proceeds from the Telethon, Généthon has become a high-tech research laboratory combining genomics, robotics and advanced computers. Généthon is currently developing strategies and tools for gene therapy. The laboratory is also reinforcing research on stem cells. Généthon is not just a supplier of vectors for laboratories via GVPN, but has the resources to cooperate in clinical research. Généthon is located in Evry, near Paris (France).
This document presents the principle of gene therapy, its aims and the tools used for gene transfer
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