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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Bibliografiske detaljer

Principais autores:	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:	Artigo
Sprog:	Inglês
Udgivet:	Elsevier 2025-06-01
Serier:	Molecular Therapy: Nucleic Acids
Fag:	MT: Oligonucleotides: Therapies and Applications oligonucleotides antisense therapeutic small molecules enhancers
Online adgang:	http://www.sciencedirect.com/science/article/pii/S2162253125000769
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