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Journal article

Induction of splice correction by cell-penetrating peptide nucleic acids.

Abstract:

BACKGROUND: Directing splicing using oligonucleotides constitutes a promising therapeutic tool for a variety of diseases such as beta-thalassemia, cystic fibrosis, and certain cancers. The rationale is to block aberrant splice sites, thus directing the splicing of the pre-mRNA towards the desired protein product. One of the difficulties in this setup is the poor bioavailability of oligonucleotides, as the most frequently used transfection agents are unsuitable for in vivo use. Here we presen...

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Publisher copy:
10.1002/jgm.950

Authors


Journal:
The journal of gene medicine
Volume:
8
Issue:
10
Pages:
1262-1273
Publication date:
2006-10-05
DOI:
EISSN:
1521-2254
ISSN:
1099-498X
URN:
uuid:ad95bd2c-9571-45ab-b16e-09b7bcd0086c
Source identifiers:
267379
Local pid:
pubs:267379

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