409. Complete Correction of Enzymatic Deficiency and Lysosomal Storage throughout the Brain in a Mouse Model of GM1-gangliosidosis after Neonatal Intracerebroventricular Injection of an AAV1 Vector Encoding Lysosomal Acid Beta- galactosidase
Broekman, Marike L. D., Baek, Rena, Comer, Laryssa A., Seyfried, Thomas N., Sena-Esteves, MiguelVolume:
13
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/j.ymthe.2006.08.472
Date:
May, 2006
File:
PDF, 82 KB
english, 2006