Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates. - Walia JS; Altaleb N; Bello A; Kruck C; LaFave MC; Varshney GK; Burgess SM; Chowdhury B; Hurlbut D; Hemming R; Kobinger GP; Triggs-Raine B (Molecular therapy : the journal of the American Society of Gene Therapy, 2015)