期刊
TRENDS IN MOLECULAR MEDICINE
卷 9, 期 6, 页码 229-232出版社
ELSEVIER SCI LTD
DOI: 10.1016/S1471-4914(03)00072-8
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资金
- Telethon [GGP02453] Funding Source: Medline
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.
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