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GENE THERAPY FOR INHERITED SEVERE PHOTORECEPTOR DISEASES (AAVEYE)
Date du début: 1 nov. 2008, Date de fin: 31 oct. 2011,
"The retina represents the visual sensory receptor of the nervous system (CNS). Inherited diseases like Retintitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA), for which no therapies are available, are due to mutations in genes preferentially expressed in the photoreceptor cells of the retina. Vectors derived from the adeno-associated virus (AAV) efficiently transduce the retina of anima ...
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