Assessment of AAV Dual Vector Safety in the Abca4(-/-) Mouse Model of Stargardt Disease

McClements, ME; Barnard, AR; Issa, PC; MacLaren, RE

MacLaren, RE (corresponding author), Univ Oxford, John Radcliffe Hosp, Nuffield Lab Ophthalmol, Clin Neurosci, Oxford OX3 9DU, England.

TRANSLATIONAL VISION SCIENCE & TECHNOLOGY, 2020; 9 (7):

Abstract

Purpose: Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ......

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