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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...

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Detalhes bibliográficos
Publicado no:J Neurosci
Main Authors: Gonzalez-Alegre, Pedro, Bode, Nicole, Davidson, Beverly L., Paulson, Henry L.
Formato: Artigo
Idioma:Inglês
Publicado em: Society for Neuroscience 2005
Assuntos:
Acesso em linha: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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