CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington’s Disease Mouse Model

Huntington’s disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin (HTT) gene, which results in the production of a mutant protein that forms inclusions and selectively destroys neurons i...

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Detalles Bibliográficos
Autores principales: Ekman, Freja K., Ojala, David S., Adil, Maroof M., Lopez, Paola A., Schaffer, David V., Gaj, Thomas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6717077/
https://www.ncbi.nlm.nih.gov/pubmed/31465962
http://dx.doi.org/10.1016/j.omtn.2019.07.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6717077/
https://www.ncbi.nlm.nih.gov/pubmed/31465962
http://dx.doi.org/10.1016/j.omtn.2019.07.009

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