Safety of intracisternal administration of AAV9 based gene therapy: Case series of PR001 in infants with type 2 Gaucher disease

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Zion Zibly, Yair Anikster, Shoshana Revel-Vilk, Ari Zimran, Mark Lowrey, Eriene A. Youssef, Jeffery Sevigny, Bryan Pukenas
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research