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Article

Gene and Cell Therapy for AIPL1-Associated Leber Congenital Amaurosis: Challenges and Prospects.

Advances in experimental medicine and biology - 1 Jan 2019

Perdigao Pedro R L, van der Spuy Jacqueline

Abstract excerpt

Leber congenital amaurosis (LCA) caused by AIPL1 mutations is one of the most severe forms of inherited retinal degeneration (IRD). The rapid and extensive photoreceptor degeneration challenges the development of potential treatments. Nevertheless, preclinical studies show that both gene augmentation and photoreceptor transplantation can regenerate and restore retinal function in animal models of AIPL1-associated...

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PMID: 31884595
DOI: 10.1007/978-3-030-27378-1_16

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