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Article

Technological advancements to study cellular signaling pathways in inherited retinal degenerative diseases.

Current opinion in pharmacology - 1 Oct 2021

Roy Akanksha, Hilhorst Riet, Groten John, Paquet-Durand François, Tomar Tushar

Abstract excerpt

Inherited retinal degenerative diseases (IRDs) are rare neurodegenerative disorders with mutations in hundreds of genes leading to vision loss, primarily owing to photoreceptor cell death. This genetic diversity is impeding development of effective treatment options. Gene-based therapies have resulted in the first FDA-approved drug (Luxturna) for RPE65-specific IRD. Although currently explored in clinical trials,...

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Identifiers

PMID: 34388439
DOI: 10.1016/j.coph.2021.07.002

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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