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Adeno-associated viral vector serotype 5 containing the human ABCA4 gene

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Company Fondazione Telethon
DescriptionAdeno-associated viral (AAV) vector serotype 5 containing the human ABCA4 gene
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard Indication Ophthalmic (unspecified)
Indication DetailsTreat Stargardt's disease
Regulatory Designation
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