Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides

Authors :: 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
Source :: Communications Biology, Vol 5, Iss 1, Pp 1-14 (2022)
Publication Year :: 2022
Publisher :: Nature Portfolio, 2022.
Abstract: 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.

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