Antisense-mediated redirection of mRNA splicing.
Antisense technology has been used to study basic biological processes, and to block these processes when they deleteriously lead to human disease. A separate, equally important application of antisense technology is to upregulate the gene expression lost in the diseased state by shifting alternative splicing of pre-messenger RNA. This strategy has commonly relied upon the use of antisense oligonucleotides; however, another approach is to use a plasmid construct to generate antisense RNA inside the cell. Antisense therapeutics based on expression vectors and viral vectors offers a gene therapy approach, whereas those based on oligonucleotides offers a more drug like approach.
Vacek
M
M
Curriculum in Genetics and Molecular Biology, University of North Carolina, Chapel Hill, North Carolina 27599, USA.
Sazani
P
P
Kole
R
R
D017398:Alternative Splicing; D000818:Animals; D016189:Dystrophin / Q000235:genetics; D005786:Gene Expression Regulation; D015316:Genetic Therapy; D005914:Globins / Q000235:genetics; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000494:pharmacology* / Q000627:therapeutic use; D012326:RNA Splicing; D016372:RNA, Antisense / Q000494:pharmacology* / Q000627:therapeutic use
D016189:Dystrophin; D016376:Oligonucleotides, Antisense; D016372:RNA, Antisense; D005914:Globins