280. Combination of Low-Dose Gene Therapy and Monthly Enzyme Replacement Therapy Improves the Phenotype of a Mouse Model of Lysosomal Storage Disease
Ferla, Rita, Alliegro, Marialuisa, Nusco, Edoardo, Claudiani, Pamela, Auricchio, AlbertoVolume:
23
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)33889-8
Date:
May, 2015
File:
PDF, 273 KB
english, 2015