Single-dose AAV9-CLN6 gene transfer slows the decline in motor and language function in variant late infantile neuronal ceroid lipofuscinosis 6: Interim results from phase 1/2 trial
Title:
Single-dose AAV9-CLN6 gene transfer slows the decline in motor and language function in variant late infantile neuronal ceroid lipofuscinosis 6: Interim results from phase 1/2 trial
Author:
de los Reyes, Emily Aylward, Shawn Meyer, Kathrin Lehwald, Lenora Albright, Charles Rogers, David L. Castelli, Jeff Jiang, Hai Goldman, Mitchell Jain, Vipul di Ronza, Alberto Barth, Jay A.