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Article

[Gene therapy for retinitis pigmentosa].

Medecine sciences : M/S - 1 Jan 2000

Ducloyer Jean-Baptiste, Le Meur Guylène, Cronin Thérèse, Adjali Oumeya, Weber Michel

Abstract excerpt

Retinitis pigmentosa is the most common blinding inherited retinal dystrophy. Gene therapy is a burgeoning revolutionary approach that paves the way to treatment of previously incurable diseases. At the end of 2017 and 2018, a gene therapy, Luxturna®, obtained a marketing authorization by respectively the FDA (Food and Drug Administration) and the EMA (European Medicines Agency). This treatment, with proven...

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PMID: 32614312
DOI: 10.1051/medsci/2020095

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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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