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 […]

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