Novel Orthogonally Hydrocarbon-Modified Cell-Penetrating Peptide Nanoparticles Mediate Efficient Delivery of Splice-Switching Antisense Oligonucleotides In Vitro and In Vivo

Novel Orthogonally Hydrocarbon-Modified Cell-Penetrating Peptide Nanoparticles Mediate Efficient Delivery of Splice-Switching Antisense Oligonucleotides In Vitro and In Vivo

Splice-switching therapy with splice-switching oligonucleotides (SSOs) has recently proven to be a clinically applicable strategy for the treatment of several mis-splice disorders. Despite this, wider application of SSOs is severely limited by the inherently poor bioavailability of SSO-based therape...

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Bibliographic Details
Main Authors: Safa Bazaz, Tõnis Lehto, Rahel Tops, Olof Gissberg, Dhanu Gupta, Burcu Bestas, Jeremy Bost, Oscar P. B. Wiklander, Helena Sork, Eman M. Zaghloul, Doste R. Mamand, Mattias Hällbrink, Rannar Sillard, Osama Saher, Kariem Ezzat, C. I. Edvard Smith, Samir EL Andaloussi, Taavi Lehto
Format: Article
Language:English
Published: MDPI AG 2021-08-01
Series:Biomedicines
Subjects:
cell-penetrating peptides
splice-switching oligonucleotides
oligonucleotide delivery
cellular uptake
endocytosis
Online Access:https://www.mdpi.com/2227-9059/9/8/1046

Internet

https://www.mdpi.com/2227-9059/9/8/1046

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