Ontology highlight
ABSTRACT:
SUBMITTER: Salegio EA
PROVIDER: S-EPMC2936496 | biostudies-literature | 2010 Sep
REPOSITORIES: biostudies-literature
Salegio E Aguilar EA Kells A P AP Richardson R M RM Hadaczek P P Forsayeth J J Bringas J J Sardi S P SP Passini M A MA Shihabuddin L S LS Cheng S H SH Fiandaca M S MS Bankiewicz K S KS
Human gene therapy 20100901 9
Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will require widespread expression of efficacious levels of acid sphingomyelinase (ASM) in the infant human brain. At present there is no treatment available for this devastating pediatric condition. This is partly because of inherent constraints associated with the efficient delivery of therapeutic agents into the CNS of higher order models. In this study we used ...[more]