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 neur...

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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.
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