Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy

Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1) 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 c...

Description complète

Enregistré dans:
Détails bibliographiques
Publié dans:Hum Gene Ther
Auteurs principaux: Gong, Yi, Berenson, Anna, Laheji, Fiza, Gao, Guangping, Wang, Dan, Ng, Carrie, Volak, Adrienn, Kok, Rene, Kreouzis, Vasileios, Dijkstra, Inge M., Kemp, Stephan, Maguire, Casey A., Eichler, Florian
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc., publishers 2019
Sujets:
Research Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6909708/
https://ncbi.nlm.nih.gov/pubmed/30358470
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.079
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires