Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities

Author:

Oikemus Sarah R.1,Pfister Edith L.2,Sapp Ellen3,Chase Kathryn O.2,Kennington Lori A.2,Hudgens Edward1,Miller Rachael2,Zhu Lihua Julie1,Chaudhary Akanksh1,Mick Eric O.4,Sena-Esteves Miguel5,Wolfe Scot A.16,DiFiglia Marian3,Aronin Neil27,Brodsky Michael H.1ORCID

Affiliation:

1. Department of Molecular Cell and Cancer Biology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA

2. Department of Medicine, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA

3. Department of Neurology, Harvard Medical School and MassGeneral Institute for Neurodegenerative Disease, Charlestown, Massachusetts, USA

4. Department of Population and Quantitative Health Sciences, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA

5. Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA

6. Department of Biochemistry and Molecular Pharmacology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA

7. RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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