CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused by CAG repeat expansion (>36 repeats) within the first exon of the huntingtin gene. Although mutant huntingtin (mHTT) is ubiquitously expressed, the brain shows robust and early degeneration. Current RNA int...

Descripción completa

Detalles Bibliográficos
Autores principales: Monteys, Alex Mas, Ebanks, Shauna A., Keiser, Megan S., Davidson, Beverly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363210/
https://www.ncbi.nlm.nih.gov/pubmed/28129107
http://dx.doi.org/10.1016/j.ymthe.2016.11.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363210/
https://www.ncbi.nlm.nih.gov/pubmed/28129107
http://dx.doi.org/10.1016/j.ymthe.2016.11.010

Ejemplares similares