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...

詳細記述

保存先:
書誌詳細
出版年:Hum Gene Ther
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc., publishers 2019
主題:
Research Articles
オンライン･アクセス: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
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