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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Detalles Bibliográficos
Autores principales: Bazaz, Safa, Lehto, Tõnis, Tops, Rahel, Gissberg, Olof, Gupta, Dhanu, Bestas, Burcu, Bost, Jeremy, Wiklander, Oscar P. B., Sork, Helena, Zaghloul, Eman M., Mamand, Doste R., Hällbrink, Mattias, Sillard, Rannar, Saher, Osama, Ezzat, Kariem, Smith, C. I. Edvard, Andaloussi, Samir EL, Lehto, Taavi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8392223/
https://www.ncbi.nlm.nih.gov/pubmed/34440250
http://dx.doi.org/10.3390/biomedicines9081046

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