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

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 p...

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Detaylı Bibliyografya
Asıl Yazarlar: El-Andaloussi, S, Johansson, H, Lundberg, P, Langel, U
Materyal Türü: Journal article
Dil:English
Baskı/Yayın Bilgisi: 2006