Article
Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human Bestrophinopathy.
Human gene therapy - 1 Sept 2026
Gray Alexa, Kwok Jennifer C, Sato Yu, Assenmacher Charles-Antoine, Choudhary Mayur, Jayagopal Ash, Hua Peiying, Ying Gui-Shuang, Swider Malgorzata, Sumaroka Alexander, Cideciyan Artur V, Gustavo Aguirre D, Beltran William A
Abstract excerpt
To treat patients affected with bestrophinopathies caused by mutations in the BEST1 gene, a recombinant adeno-associated virus vector (OPGx-BEST1 or rAAV2/2-VMD2-BEST1) is being developed. The vector construct includes the human BEST1 cDNA under control of the VMD2/BEST1 promoter and is packaged in an AAV2 capsid. This study evaluated the efficacy and toxicity of OPGx-BEST1 administered by subretinal injection in...
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