Cell-penetrating peptides-based strategies for the delivery of splice redirecting antisense oligonucleotides.
Methods Mol Biol
; 764: 75-89, 2011.
Article
em En
| MEDLINE
| ID: mdl-21748634
ABSTRACT
Progress in our understanding of the molecular pathogenesis of human malignancies has provided therapeutic targets amenable to oligonucleotide (ON)-based strategies. Antisense ON-mediated splicing regulation in particular offers promising prospects since the majority of human genes undergo alternative splicing and since splicing defects have been found in many diseases. However, their implementation has been hampered so far by the poor bioavailability of nucleic acids-based drugs. Cell-penetrating peptides (CPPs) now appear as promising non-viral delivery vector for non-permeant biomolecules. We describe here new CPPs allowing the delivery of splice redirecting steric-block ON using either chemical conjugation or non-covalent complexation. We also describe a convenient and robust splice redirecting assay which allows the quantitative assessment of ON nuclear delivery.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Bioensaio
/
RNA Mensageiro
/
Oligonucleotídeos Antissenso
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Sistemas de Liberação de Medicamentos
/
Processamento Alternativo
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Substâncias Macromoleculares
/
Peptídeos Penetradores de Células
Limite:
Female
/
Humans
Idioma:
En
Revista:
Methods Mol Biol
Assunto da revista:
BIOLOGIA MOLECULAR
Ano de publicação:
2011
Tipo de documento:
Article
País de afiliação:
Suécia