Replacement of huntingtin exon 1 by trans-splicing

Huntington’s disease (HD) is an autosomal-dominant neurodegenerative disorder caused by polyglutamine expansion in the amino-terminus of huntingtin (HTT). HD offers unique opportunities for promising RNA-based therapeutic approaches aimed at reducing mutant HTT expression, since the HD mutation is c...

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Autores principales: Rindt, Hansjörg, Yen, Pei-Fen, Thebeau, Christina N., Peterson, Troy S., Weisman, Gary A., Lorson, Christian L.
Formato: Artigo
Lenguaje:Inglês
Publicado: SP Birkhäuser Verlag Basel 2012
Materias:
Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3874886/
https://ncbi.nlm.nih.gov/pubmed/22814437
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00018-012-1083-5
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