Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

siRNAs comprise a class of drugs that can be programmed to silence any target gene. Chemical engineering efforts resulted in development of divalent siRNAs (di-siRNAs), which support robust and long-term efficacy in rodent and nonhuman primate brains upon direct cerebrospinal fluid (CSF) administrat...

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Detalles Bibliográficos
Autores principales: Ferguson, Chantal M., Godinho, Bruno M.D.C., Alterman, Julia F., Coles, Andrew H., Hassler, Matthew, Echeverria, Dimas, Gilbert, James W., Knox, Emily G., Caiazzi, Jillian, Haraszti, Reka A., King, Robert M., Taghian, Toloo, Puri, Ajit, Moser, Richard P., Gounis, Matthew J., Aronin, Neil, Gray-Edwards, Heather, Khvorova, Anastasia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Resource and Technical Advance
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8783676/
https://www.ncbi.nlm.nih.gov/pubmed/34935646
http://dx.doi.org/10.1172/jci.insight.152203

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