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01-03-2018 | Review Article

Leber’s Congenital Amaurosis and Gene Therapy

Authors: Brijesh Takkar, Pooja Bansal, Pradeep Venkatesh

Published in: Indian Journal of Pediatrics | Issue 3/2018

Abstract

Retinal blindness is an important cause of pediatric visual loss. Leber’s congenital amaurosis (LCA) is one of these causes, often wrongly included in the spectrum of retinitis pigmentosa. The disease has become the center of research after initial reports of success in management with gene therapy. This review discusses in brief the clinical presentation and investigative modalities used in LCA. Further, the road to gene discovery and details of currently applied gene therapy are presented. LCA is one of the first successfully managed human diseases and offers an entirely new dimension in ocular therapeutics.

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Title: Leber’s Congenital Amaurosis and Gene Therapy
Authors: Brijesh Takkar
Pooja Bansal
Pradeep Venkatesh
Publication date: 01-03-2018
Publisher: Springer India
Published in: Indian Journal of Pediatrics / Issue 3/2018
Print ISSN: 0019-5456
Electronic ISSN: 0973-7693
DOI: https://doi.org/10.1007/s12098-017-2394-1

Keynote webinar | Spotlight on sleep in brain health

Springer Medicine

Leber’s Congenital Amaurosis and Gene Therapy

Abstract

Keynote webinar | Spotlight on sleep in brain health

Springer Medicine

Abstract

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