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RAAV2

Gene therapy

decarboxylase deficiency. It uses a recombinant adeno-associated virus serotype 2 (rAAV2) to deliver a functioning DOPA decarboxylase (DDC) gene directly into the

Sangamo Therapeutics

clinicaltrials.gov. Retrieved July 28, 2020. "Dose-Ranging Study of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

Adeno-associated virus

2003). "Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques". Molecular Therapy. 8 (6): 918–26. doi:10.1016/j

Leber's hereditary optic neuropathy

administration of GS010, a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing human wildtype mitochondrial NADH dehydrogenase 4 (ND4) gene

Gene therapy for osteoarthritis

gov Clinical trial number NCT02790723 for "Safety of Intra-Articular Sc-rAAV2.5IL-1Ra in Subjects With Moderate Knee OA (AAVIL-1Ra)" at ClinicalTrials