X-Message-Number: 24994
From: "The NanoAging Institute" <>

Subject: Great News: Development of an Innovative Technique of Gene Therapy: 
Exon Skipping
Date: Mon, 8 Nov 2004 04:50:39 -0500

PARIS, France, November 4 /PRNewswire/ -- A team of researchers at Genethon, 
the laboratory created and financed by the Association Francaise contre les 
Myopathies (AFM) thanks to the French Telethon donations, has succeeded in 
repairing the muscles of mice models of Duchenne muscular dystrophy thanks 
to a gene therapy called 'exon skipping'. The exon skip occurs during the 
intermediate phase between the gene and the protein at the moment of 
splicing (1), and allows production of a truncated but functional protein to 
be restored. This advance illustrates the progress made in gene therapy 
techniques over the last ten years. By now, these techniques have become 
very sophisticated and, by intervening directly on the message of the gene, 
they open new therapeutic prospects for genetic diseases. This work was 
carried out by a Genethon team led by Olivier Danos and Luis Garcia (CNRS 
UMR 8115) in collaboration with researchers at the Cochin Institute in 
Paris. It is published today by the magazine Science in its online edition 
Science Express.


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