Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides

The therapeutic and research potentials of oligonucleotides (ONs) have been hampered in part by their inability to effectively escape endosomal compartments to reach their cytosolic and nuclear targets. Splice-switching ONs (SSOs) can be used with endosomolytic small molecule compounds to increase f...

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Detalles Bibliográficos
Autores principales: Bost, Jeremy P., Ojansivu, Miina, Munson, Michael J., Wesén, Emelie, Gallud, Audrey, Gupta, Dhanu, Gustafsson, Oskar, Saher, Osama, Rädler, Julia, Higgins, Stuart G., Lehto, Taavi, Holme, Margaret N., Dahlén, Anders, Engkvist, Ola, Strömstedt, Per-Erik, Andersson, Shalini, Edvard Smith, C. I., Stevens, Molly M., Esbjörner, Elin K., Collén, Anna, El Andaloussi, Samir
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/PMC8888659/
https://www.ncbi.nlm.nih.gov/pubmed/35233031
http://dx.doi.org/10.1038/s42003-022-03132-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8888659/
https://www.ncbi.nlm.nih.gov/pubmed/35233031
http://dx.doi.org/10.1038/s42003-022-03132-2

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