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.
| Main Authors: | , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2022-10-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-022-33061-x |
| _version_ | 1828106184054276096 |
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| 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 Edith L. Pfister |
| author_facet | 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 |
| author_sort | Faith Conroy |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-11T10:13:09Z |
| format | Article |
| id | doaj.art-b3726cec35ca4048a177c94f5d18c4bd |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-04-11T10:13:09Z |
| publishDate | 2022-10-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-b3726cec35ca4048a177c94f5d18c4bd2022-12-22T04:30:03ZengNature PortfolioNature Communications2041-17232022-10-0113111410.1038/s41467-022-33061-xChemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease modelsFaith Conroy0Rachael Miller1Julia F. Alterman2Matthew R. Hassler3Dimas Echeverria4Bruno M. D. C. Godinho5Emily G. Knox6Ellen Sapp7Jaquelyn Sousa8Ken Yamada9Farah Mahmood10Adel Boudi11Kimberly Kegel-Gleason12Marian DiFiglia13Neil Aronin14Anastasia Khvorova15Edith L. Pfister16Department of Medicine, UMass Chan Medical SchoolDepartment of Medicine, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolDepartment of Neurology, Massachusetts General Hospital, Harvard Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolDepartment of Neurology, Massachusetts General Hospital, Harvard Medical SchoolDepartment of Neurology, Massachusetts General Hospital, Harvard Medical SchoolDepartment of Neurology, Massachusetts General Hospital, Harvard Medical SchoolDepartment of Neurology, Massachusetts General Hospital, Harvard Medical SchoolDepartment of Medicine, UMass Chan Medical SchoolRNA Therapeutics Institute, UMass Chan Medical SchoolDepartment of Medicine, UMass Chan Medical SchoolChemically 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.https://doi.org/10.1038/s41467-022-33061-x |
| spellingShingle | 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 Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models Nature Communications |
| title | Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models |
| title_full | Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models |
| title_fullStr | Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models |
| title_full_unstemmed | Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models |
| title_short | Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models |
| title_sort | chemical engineering of therapeutic sirnas for allele specific gene silencing in huntington s disease models |
| url | https://doi.org/10.1038/s41467-022-33061-x |
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