about
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapyInsulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathyProgressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.
P50
description
investigador
@es
researcher
@en
wetenschapper
@nl
name
Sandra Motas
@en
Sandra Motas
@nl
type
label
Sandra Motas
@en
Sandra Motas
@nl
prefLabel
Sandra Motas
@en
Sandra Motas
@nl
P31
P496
0000-0003-2748-5952