Lahey HG, Webber CJ, Golebiowski D, Izzo CM, Horn E, Taghian T, Rodriguez P, Batista AR, Ellis LE, Hwang M, Martin DR, Gray-Edwards H, Sena-Esteves M. Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice. Mol Ther. 2020 10 07; 28(10):2150-2160.