GENE THERAPY FULLY RESTORES VISION IN MOUSE MODEL OF LEBER CONGENITAL AMAUROSIS (TERAPIA GÉNICA RESTABLECE VISIÓN EN RATONES CON ENF. LEBER)

Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success of this approach strongly support clinical testing of a […]

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