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

Abstract:

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 experimental models. However, translational approaches are required to transform SCOs into usable drug products. In this study, we present a new cell-penetrating peptide, PepFect14 (PF14), which efficiently...

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Publication status:
Published

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Publisher copy:
10.1093/nar/gkr072

Authors


Andaloussi, SE More by this author
Zaghloul, EM More by this author
Lindberg, S More by this author
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Journal:
Nucleic acids research
Volume:
39
Issue:
12
Pages:
5284-5298
Publication date:
2011-07-05
DOI:
EISSN:
1362-4962
ISSN:
0305-1048
URN:
uuid:e37bead1-caf8-4031-ab8e-d89bd1749878
Source identifiers:
120161
Local pid:
pubs:120161

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