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

Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimized to provide efficient delivery to the CNS, enabling development of CNS-targeted therapeu...

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Detalles Bibliográficos
Autores principales: Conroy, Faith, Miller, Rachael, Alterman, Julia F., Hassler, Matthew R., Echeverria, Dimas, Godinho, Bruno M. D. C., Knox, Emily G., Sapp, Ellen, Sousa, Jaquelyn, Yamada, Ken, Mahmood, Farah, Boudi, Adel, Kegel-Gleason, Kimberly, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Pfister, Edith L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9530163/
https://www.ncbi.nlm.nih.gov/pubmed/36192390
http://dx.doi.org/10.1038/s41467-022-33061-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9530163/
https://www.ncbi.nlm.nih.gov/pubmed/36192390
http://dx.doi.org/10.1038/s41467-022-33061-x

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