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

• Main Authors: Freja K. Ekman (Author), David S. Ojala (Author), Maroof M. Adil (Author), Paola A. Lopez (Author), David V. Schaffer (Author), Thomas Gaj (Author)
• Format: Book
• Published: Elsevier, 2019-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 neur...