Leber Congenital Amaurosis News and Research

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FDA-approved compound promotes photoreceptor survival in lab models of inherited retinal ciliopathy

A National Eye Institute-led team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA 10), an inherited retinal ciliopathy disease that often results in severe visual impairment or blindness in early childhood.

30 Mar 2023

Genome editing proven effective to restore the vision of mice with genetic diseases

Chinese researchers have turned out to be successful in restoring the vision of mice with retinitis pigmentosa, which is considered to be one of the significant causes of blindness in humans.

20 Mar 2023

Adults with congenital blindness experience striking recoveries of night vision after gene therapy

Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.

11 Oct 2022

Leber Congenital Amaurosis News and Research

FDA-approved compound promotes photoreceptor survival in lab models of inherited retinal ciliopathy

Genome editing proven effective to restore the vision of mice with genetic diseases

Adults with congenital blindness experience striking recoveries of night vision after gene therapy

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