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

Autor: 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
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/s41467-022-33061-x
Popis: 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.
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