Ontology highlight
ABSTRACT:
SUBMITTER: Gong Y
PROVIDER: S-EPMC6909708 | biostudies-literature | 2019 May
REPOSITORIES: biostudies-literature
Gong Yi Y Berenson Anna A Laheji Fiza F Gao Guangping G Wang Dan D Ng Carrie C Volak Adrienn A Kok Rene R Kreouzis Vasileios V Dijkstra Inge M IM Kemp Stephan S Maguire Casey A CA Eichler Florian F
Human gene therapy 20181218 5
Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (<i>ABCD1</i>) cause elevations in very long-chain fatty acids (VLCFAs) and the neurodegenerative disease adrenoleukodystrophy (ALD). In most adults, this manifests as the spinal cord axonopathy adrenomyeloneuropathy (AMN). A challenge in virus-based gene therapy in AMN is how to achieve functional gene correction to the entire spinal cord while minimizing leakage into the systemic circulation, which could contribute ...[more]