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: Yamada, Ken, Hariharan, Vignesh Narayan, Caiazzi, Jillian, Miller, Rachael, Furguson, Chantal, Sapp, Ellen, Fakih, Hassan, Tan, Qi, Yamada, Nozomi, Furgal, Raymond C., Paquette, Joseph, Bramato, Brianna, McHugh, Nicholas, Summers, Ashley, Lochmann, Clemens, Godinho, Bruno M.D.C., Hildebrand, Samuel, Echeverria, Dimas, Hassler, Matthew R., Alterman, Julia F., DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10245983/
https://www.ncbi.nlm.nih.gov/pubmed/37292886
http://dx.doi.org/10.1101/2023.05.26.542506
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