Available Evidence on Leber Congenital Amaurosis and Gene Therapy

Alkharashi, M; Fulton, AB

Alkharashi, M (reprint author), Harvard Med Sch, Boston Childrens Hosp, Dept Ophthalmol, Pediat Ophthalmol & Strabismus, 300 Longwood Ave,Fegan 4, Boston, MA 02115 USA.

SEMINARS IN OPHTHALMOLOGY, 2017; 32 (1): 14

Abstract

Leber congenital amaurosis (LCA) is a group of severe inherited retinal dystrophies that lead to early childhood blindness. In the last decade, intere......

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