Serge Braun

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letter to the editor large budgets by various government agencies that have permitted intense collaboration among scientists as well as engagement of industry for the development of supporting technologies. The funding made available for these projects contrasts sharply with the relatively limited budgets that have been available for gene therapy research.(More)
A phase I open clinical study on gene therapy in Duchenne and Becker muscular dystrophy, without direct individual benefit for the patient, is being performed at the Pitié-Salpêtrière Hospital, Paris. The aims of this project are: (a) to determine the tolerance and the safety of the intramuscular administration of dystrophin cDNA and (b) to study the(More)
Since the identification of abnormalities in the dystrophin gene as primary cause of Duchenne muscular dystrophy, gene therapy has been seen as an obvious option among various approaches to treat the disease. It is also considered to be especially challenging, as in this context, one must achieve massive transfer of the gene with a sustained lifelong(More)
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