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PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation

Numerous human genetic diseases are caused by mutations that give rise to aberrant alternative splicing. Recently, several of these debilitating disorders have been shown to be amenable for splice-correcting oligonucleotides (SCOs) that modify splicing patterns and restore the phenotype in experimen...

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Detalles Bibliográficos
Autores principales: Ezzat, Kariem, EL Andaloussi, Samir, Zaghloul, Eman M., Lehto, Taavi, Lindberg, Staffan, Moreno, Pedro M. D., Viola, Joana R., Magdy, Tarek, Abdo, Rania, Guterstam, Peter, Sillard, Rannar, Hammond, Suzan M., Wood, Matthew J. A., Arzumanov, Andrey A., Gait, Michael J., Smith, C. I. Edvard, Hällbrink, Mattias, Langel, Ülo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2011
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3130259/
https://www.ncbi.nlm.nih.gov/pubmed/21345932
http://dx.doi.org/10.1093/nar/gkr072