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Article

The new landscape of retinal gene therapy.

American journal of medical genetics. Part C, Seminars in medical genetics - 1 Sept 2020

Ku Cristy A, Pennesi Mark E

Abstract excerpt

Novel therapeutics for inherited retinal dystrophies (IRDs) have rapidly evolved since groundbreaking clinical trials for LCA due to RPE65 mutations led to the first FDA-approved in vivo gene therapy. Since then, advancements in viral vectors have led to more efficient AAV transduction and developed other viral vectors for gene augmentation therapy of large gene targets. Furthermore, significant developments in...

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Identifiers

PMID: 32888388
DOI: 10.1002/ajmg.c.31842

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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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