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...

Cur síos iomlán

Sonraí bibleagrafaíochta
Príomhchruthaitheoirí: El-Andaloussi, S, Johansson, H, Lundberg, P, Langel, U
Formáid: Journal article
Teanga:English
Foilsithe / Cruthaithe: 2006