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Start Over Author "Farah Mahmood" Journal nature communications
1 results on '"Farah Mahmood"'

1. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

Author

Faith Conroy, Rachael Miller, Julia F. Alterman, Matthew R. Hassler, Dimas Echeverria, Bruno M. D. C. Godinho, Emily G. Knox, Ellen Sapp, Jaquelyn Sousa, Ken Yamada, Farah Mahmood, Adel Boudi, Kimberly Kegel-Gleason, Marian DiFiglia, Neil Aronin, Anastasia Khvorova, and Edith L. Pfister

Subjects
Science
Abstract

Chemically modified siRNAs distinguish between mutant and normal huntingtin based on a single nucleotide difference and lower mutant huntingtin specifically in patient derived cells and in a mouse model of Huntington’s disease.

Published
2022
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