Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon.

Surono, Agus

Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon. [electronic resource] - Human gene therapy Aug 2004 - 749-57 p. digital

Publication Type: Case Reports; Comparative Study; Journal Article; Research Support, Non-U.S. Gov't

1043-0342

10.1089/1043034041648444 doi


Base Sequence
Blotting, Western
Codon, Nonsense--genetics
DNA Primers
Dystrophin--genetics
Ethylenes--chemistry
Exons--genetics
Genetic Therapy--methods
Humans
Immunohistochemistry
Muscle Fibers, Skeletal--metabolism
Muscular Dystrophy, Duchenne--metabolism
Oligonucleotides, Antisense--genetics
RNA--chemistry
RNA Editing--genetics
RNA, Messenger--genetics
Reverse Transcriptase Polymerase Chain Reaction
Transcription, Genetic--drug effects
Transfection