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Optimization of trans-Splicing for Huntington's Disease RNA Therapy

Huntington's disease (HD) is a devastating neurodegenerative disorder caused by a polyglutamine (polyQ) expansion in exon 1 of the Huntingtin (HTT) gene. We have previously demonstrated that spliceosome-mediated trans-splicing is a viable molecular strategy to specifically reduce and repair mut...

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Detalles Bibliográficos
Autores principales:	Rindt, Hansjörg, Tom, Colton M., Lorson, Christian L., Mattis, Virginia B.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2017
Materias:	Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5641306/ https://www.ncbi.nlm.nih.gov/pubmed/29066943 http://dx.doi.org/10.3389/fnins.2017.00544

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