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Article

Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies.

Gene therapy - 1 Jul 2026

Marie Mélanie, Churet Lucie, Gautron Anne-Sophie, Farjo Rafal, Mizuyoshi Kensuke, Stevenson Victoria, Khabou Hanen, Léveillard Thierry, Sahel José-Alain, Lorget Florence

Abstract excerpt

Rod-cone dystrophies (RCD) are caused by mutations in over 100 genes associated with photoreceptor function, leading to progressive and sequential loss of rod and cone photoreceptors. These mutations generally disrupt retinal metabolism and oxidative stress response accelerating disease progression and vision loss. SPVN06 is an adeno-associated virus (AAV)-based gene- and mutation-agnostic investigational therapy...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 40759732
DOI: 10.1038/s41434-025-00556-3
PMCID: PMC13473012

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

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