Journal
TRENDS IN MOLECULAR MEDICINE
Volume 9, Issue 6, Pages 229-232Publisher
ELSEVIER SCI LTD
DOI: 10.1016/S1471-4914(03)00072-8
Keywords
-
Funding
- Telethon [GGP02453] Funding Source: Medline
Ask authors/readers for more resources
It is becoming clear that exonic sequences can act as determinants of their own fate: the inclusion or exclusion from mature mRNA. Indeed, even silent nucleotide substitutions can cause aberrant exon skipping, resulting in a disease phenotype. It might be possible to restore essential splicing functions, lost through mutations, using molecular therapy at the RNA level. A variety of methods have been attempted, the most promising being the recent use of chimeric compounds that localize splicing-functional peptides by base complementarity.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available