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...
Main Authors: | Freja K. Ekman, David S. Ojala, Maroof M. Adil, Paola A. Lopez, David V. Schaffer, Thomas Gaj |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2019-09-01
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Series: | Molecular Therapy: Nucleic Acids |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2162253119301994 |
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