Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides

Autor: Jeremy P. Bost, Miina Ojansivu, Michael J. Munson, Emelie Wesén, Audrey Gallud, Dhanu Gupta, Oskar Gustafsson, Osama Saher, Julia Rädler, Stuart G. Higgins, Taavi Lehto, Margaret N. Holme, Anders Dahlén, Ola Engkvist, Per-Erik Strömstedt, Shalini Andersson, C. I. Edvard Smith, Molly M. Stevens, Elin K. Esbjörner, Anna Collén, Samir El Andaloussi
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Communications Biology, Vol 5, Iss 1, Pp 1-14 (2022)
Druh dokumentu: article
ISSN: 2399-3642
DOI: 10.1038/s42003-022-03132-2
Popis: Two new endosomolytic small compounds increase delivery of splice-switching oligonucleotides by interfering with their trafficking to the LAMP1-positive endosomal compartments, inducing endosomal membrane rupture and concurrent oligonucleotide escape into the cytosol.
Databáze: Directory of Open Access Journals
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