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

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

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.

Bibliographic Details
Main Authors: 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, Edith L. Pfister
Format: Article
Language:English
Published: Nature Portfolio 2022-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-33061-x

Internet

https://doi.org/10.1038/s41467-022-33061-x

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