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Article

AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2018

Alves C Henrique, Wijnholds Jan

Abstract excerpt

Mutations in the CRB1 gene account for around 10,000 persons with Leber congenital amaurosis (LCA) and 70,000 persons with retinitis pigmentosa (RP) worldwide. Therefore, the CRB1 gene is a key target in the fight against blindness. A proof-of-concept for an adeno-associated virus (AAV)-mediated CRB2 gene augmentation therapy for CRB1-RP was recently described. Preclinical studies using animal models such as...

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Source

Identifiers

PMID: 29188511
DOI: 10.1007/978-1-4939-7522-8_10

Genes

CRB1
CRB2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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