Ontology highlight
ABSTRACT:
SUBMITTER: Ruzo A
PROVIDER: S-EPMC3278559 | biostudies-literature | 2012 Feb
REPOSITORIES: biostudies-literature
Ruzo Albert A Garcia Miquel M Ribera Albert A Villacampa Pilar P Haurigot Virginia V Marcó Sara S Ayuso Eduard E Anguela Xavier M XM Roca Carles C Agudo Judith J Ramos David D Ruberte Jesús J Bosch Fatima F
Molecular therapy : the journal of the American Society of Gene Therapy 20111018 2
Mucopolysaccharidosis type IIIA (MPSIIIA) is an inherited lysosomal storage disease caused by deficiency of sulfamidase, resulting in accumulation of the glycosaminoglycan (GAG) heparan sulfate. It is characterized by severe progressive neurodegeneration, together with somatic alterations, which lead to death during adolescence. Here, we tested the ability of adeno-associated virus (AAV) vector-mediated genetic modification of either skeletal muscle or liver to revert the already established dis ...[more]