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Silencing Primary Dystonia: Lentiviral-Mediated RNA Interference Therapy for DYT1 Dystonia

DYT1 is the most common inherited dystonia. Currently, there are no preventive or curative therapies for this dominantly inherited disease. DYT1 dystonia is caused by a common three-nucleotide deletion in the TOR1A gene that eliminates a glutamic acid residue from the protein torsinA. Recent studies...

詳細記述

保存先:
書誌詳細
出版年:J Neurosci
主要な著者: Gonzalez-Alegre, Pedro, Bode, Nicole, Davidson, Beverly L., Paulson, Henry L.
フォーマット: Artigo
言語:Inglês
出版事項: Society for Neuroscience 2005
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6725832/
https://ncbi.nlm.nih.gov/pubmed/16280588
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.3016-05.2005
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