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Article

A synthetic opsin restores vision in patients with severe retinal degeneration.

Molecular therapy : the journal of the American Society of Gene Therapy - 7 May 2025

Mohanty Samarendra K, Mahapatra Santosh, Batabyal Subrata, Carlson Michael, Kanungo Gayatri, Ayyagari Ananta, Tchedre Kissaou, Franco Joel A, Singer Michael, Barone Samuel B, Chavala Sai, Mahajan Vinit B

Abstract excerpt

Inherited retinal degenerations are the leading cause of blindness worldwide, and, in advanced stages, cell loss makes gene replacement ineffective. Optogenetics offers a therapeutic opportunity to restore vision by photo-sensitizing remaining retinal neurons. However, current opsins are kinetically slow, partially activated in ambient light, unresponsive to different light colors, and target low-resolution...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 40121528
DOI: 10.1016/j.ymthe.2025.03.031
PMCID: PMC12126831

Genes

ABCA4

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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