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...

詳細記述

書誌詳細

主要な著者:	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
フォーマット:	Artigo
言語:	Inglês
出版事項:	Elsevier 2025-06-01
シリーズ:	Molecular Therapy: Nucleic Acids
主題:	MT: Oligonucleotides: Therapies and Applications oligonucleotides antisense therapeutic small molecules enhancers
オンライン･アクセス:	http://www.sciencedirect.com/science/article/pii/S2162253125000769
タグ:	タグ追加 タグなし, このレコードへの初めてのタグを付けませんか!