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Design of 2'-O-Me RNA/ENA chimera oligonucleotides to induce exon skipping in dystrophin pre-mRNA.
- Source :
-
Nucleic acids symposium series (2004) [Nucleic Acids Symp Ser (Oxf)] 2004 (48), pp. 297-8. - Publication Year :
- 2004
-
Abstract
- 2'-O-Me RNA/ENA chimera oligonucleotides complementary to exon 45 and 46 of the dystrophin gene induced exon 45 and 46 skipping of the dystrophin pre-mRNA, respectively. The induction of exon skipping by the most effective 2'-O-Me RNA/ENA chimeras led to the expression of dystrophin in dystrophin-deficient myocytes by correcting the translational reading frames. Also, in the process of 2'-O-Me RNA/ENA chimera optimization to induce exon skipping in several exons, it was found that the optimized target sequences of the chimeras included guanosine- or adenosine-rich sequences that might function as spiking enhancer sequences (SES).
- Subjects :
- Adenosine
Base Sequence
Drug Design
Dystrophin metabolism
Gene Expression Regulation
Guanosine
Humans
Nucleic Acid Heteroduplexes chemistry
Nucleic Acids chemistry
Oligonucleotides chemistry
RNA chemistry
Dystrophin genetics
Exons genetics
Nucleic Acid Heteroduplexes metabolism
Nucleic Acids metabolism
Oligonucleotides metabolism
RNA metabolism
RNA Precursors genetics
Subjects
Details
- Language :
- English
- ISSN :
- 1746-8272
- Issue :
- 48
- Database :
- MEDLINE
- Journal :
- Nucleic acids symposium series (2004)
- Publication Type :
- Academic Journal
- Accession number :
- 17150596
- Full Text :
- https://doi.org/10.1093/nass/48.1.297