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...

Descripción completa

Guardado en:
Detalles Bibliográficos
Publicado en:Hum Gene Ther
Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc., publishers 2019
Materias:
Research Articles
Acceso en línea: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
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares