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Article

AAV8-Mediated Gene Therapy Rescues Retinal Degeneration Phenotype in a Tlcd3b Knockout Mouse Model.

Investigative ophthalmology & visual science - 2 Mar 2022

Qian Xinye, Liu Hehe, Fu Shangyi, Lu Jiaxiong, Hung Yu-Ting, Turner Cassidy, Gu Haiwei, Chen Rui

Abstract excerpt

Purpose: The purpose of this study was to assess the therapeutic efficacy of rAAV8-hGRK1-Tlcd3b in a Tlcd3b-/- mouse model of retinal generation and validate TLCD3B's role as a ceramide synthase in vivo. Methods: Using Tlcd3b-/- mice as an inherited retinal disease animal model, we performed subretinal injection of rAAV8-hGRK1-Tlcd3b and evaluated the efficacy of gene replacement therapy. Tlcd3b-/- mice were...

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Identifiers

PMID: 35275174
DOI: 10.1167/iovs.63.3.11
PMCID: PMC8934561

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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