Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo

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Metabolic stabilization of therapeutic oligonucleotides requires both sugar and backbone modifications, where phosphorothioate (PS) is the only backbone chemistry used in the clinic. Here, we describe the discovery, synthesis, and characterization of a novel biologically compatible backbone, extende...

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Detalles Bibliográficos
Autores principales: Hariharan, Vignesh Narayan, Caiazzi, Jillian, Miller, Rachael, Ferguson, Chantal, Sapp, Ellen, Fakih, Hassan, Tang, Qi, Yamada, Nozomi, Furgal, Raymond, Paquette, Joseph, Bramato, Brianna, McHugh, Nicholas, Summers, Ashley, Lochmann, Clemens, Godinho, Bruno, Hildebrand, Samuel, Echeverria, Dimas, Hassler, Matthew, Alterman, Julia, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Yamada, Ken
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Journal Experts 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10312934/
https://www.ncbi.nlm.nih.gov/pubmed/37398145
http://dx.doi.org/10.21203/rs.3.rs-2987323/v1
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