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AAV8 vector encoding GUCY2D gene

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Company Fondazione Telethon
DescriptionAdeno-associated virus (AAV) serotype 8 vector encoding the guanylate cyclase 2D membrane retina-specific (GUCY2D) gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector
Latest Stage of DevelopmentPreclinical
Standard IndicationBlindness
Indication DetailsTreat Leber's congenital amaurosis (LCA)
Regulatory Designation EU - Orphan Drug (Treat Leber's congenital amaurosis (LCA))

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