Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotides in vitro

Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotides in vitro

Antisense oligonucleotides (ASOs), including splice-switching oligonucleotides (SSOs), are promising therapeutic approaches for targeting genetic defects. ASOs act in the nucleus and the cytosol to cleave mRNAs via the RNaseH1 mechanism (e.g., gapmers), while SSOs alter transcript splicing to restor...

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Main Authors: H. Yesid Estupiñán, Tom Baladi, Samantha Roudi, Michael J. Munson, Jeremy Bost, Oskar Gustafsson, Daniel Velásquez-Ramírez, Deepak Kumar Bhatt, Daniel Hagey, Dennis Hekman, Shalini Andersson, Samir EL Andaloussi, Anders Dahlén
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
oligonucleotides
antisense
therapeutic
small molecules
enhancers
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125000769
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