EYE TREATMENT CLOSES IN ON BEING THE FIRST GENE THERAPY APPROVED IN U.S.A

What could become the first gene therapy to win approval in the United States moved closer to market on Monday, when its developer announced that the medicine had succeeded in a late-stage clinical trial in treating an inherited eye disease that can cause blindness. The developer, Spark Therapeutics, said the treatment had allowed people with […]

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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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STARGARDT APNES ARGENTINA SOLICITA PACIENTES CON COROIDEREMIA Y AMAUROSIS CONGÉNITA DE LEBER

Atención amigos con COROIDEREMIA y AMAUROSIS CONGÉNITA DE LEBER: La Dra. Natalia Fernandez Fruttero está buscando los casos de COROIDEREMIA y de AMAUROSIS CONGÉNITA DE LEBER que se encuentren en Latinoamérica. Ella está trabajando para el grupo científico de Jean Bennett en la Universidad de Pennsylvania que desarrolló el tratamiento que le devolvió la vista […]

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