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

• 主要な著者: Freja K. Ekman (著者), David S. Ojala (著者), Maroof M. Adil (著者), Paola A. Lopez (著者), David V. Schaffer (著者), Thomas Gaj (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2019-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: 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...