Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene

Juvenile neuronal ceroid lipofuscinosis (JNCL or CLN3 disease) is an autosomal recessive lysosomal storage disease resulting from mutations in the CLN3 gene that encodes a lysosomal membrane protein. The disease primarily affects the brain with widespread intralysosomal accumulation of autofluoresce...

詳細記述

保存先:
書誌詳細
主要な著者: Sondhi, Dolan, Scott, Emma C., Chen, Alvin, Hackett, Neil R., Wong, Andrew M.S., Kubiak, Agnieszka, Nelvagal, Hemanth R., Pearse, Yewande, Cotman, Susan L., Cooper, Jonathan D., Crystal, Ronald G.
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc. 2014
主題:
Research Articles
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC3955974/
https://ncbi.nlm.nih.gov/pubmed/24372003
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.253
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