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...
প্রধান লেখক: | , , , |
---|---|
বিন্যাস: | Journal article |
ভাষা: | English |
প্রকাশিত: |
2006
|