Assessment of AAV Dual Vector Safety in theAbca4^-/- Mouse Model of Stargardt Disease.

ABSTRACT:

Purpose

Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ATP-binding cassette transporter family member gene (ABCA4). The nature of the dual vector system creates the potential for adverse events. Here we have investigated an overlapping adeno-associated viral ABCA4 dual vector system for signs of toxicity in Abca4^-/- mice as a prelude to dual vector first in human clinical trials.

Methods

Abca4^-/- mice received a subretinal injection of a 1:1 5':3' dual vector mix; 5' vector only; 3 ' vector only; a GFP reporter vector; or diluent only (sham). All vectors were adeno-associated virus-8 Y733F. Mice were subsequently assessed for signs o

SUBMITTER: McClements ME 

PROVIDER: S-EPMC7115835 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature