Nature Communications (Oct 2022)
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
- 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
Affiliations
- Faith Conroy
- Department of Medicine, UMass Chan Medical School
- Rachael Miller
- Department of Medicine, UMass Chan Medical School
- Julia F. Alterman
- RNA Therapeutics Institute, UMass Chan Medical School
- Matthew R. Hassler
- RNA Therapeutics Institute, UMass Chan Medical School
- Dimas Echeverria
- RNA Therapeutics Institute, UMass Chan Medical School
- Bruno M. D. C. Godinho
- RNA Therapeutics Institute, UMass Chan Medical School
- Emily G. Knox
- RNA Therapeutics Institute, UMass Chan Medical School
- Ellen Sapp
- Department of Neurology, Massachusetts General Hospital, Harvard Medical School
- Jaquelyn Sousa
- RNA Therapeutics Institute, UMass Chan Medical School
- Ken Yamada
- RNA Therapeutics Institute, UMass Chan Medical School
- Farah Mahmood
- Department of Neurology, Massachusetts General Hospital, Harvard Medical School
- Adel Boudi
- Department of Neurology, Massachusetts General Hospital, Harvard Medical School
- Kimberly Kegel-Gleason
- Department of Neurology, Massachusetts General Hospital, Harvard Medical School
- Marian DiFiglia
- Department of Neurology, Massachusetts General Hospital, Harvard Medical School
- Neil Aronin
- Department of Medicine, UMass Chan Medical School
- Anastasia Khvorova
- RNA Therapeutics Institute, UMass Chan Medical School
- Edith L. Pfister
- Department of Medicine, UMass Chan Medical School
- DOI
- https://doi.org/10.1038/s41467-022-33061-x
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 14
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.
