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

Internet

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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