Less is more: therapeutic exon skipping for Duchenne muscular dystrophy.
Aartsma-Rus
Annemieke
A
van Ommen
Gert-Jan B
GJ
D000818:Animals; D016189:Dystrophin / Q000235:genetics; D005091:Exons; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*
C484258:DMD protein, human; D016189:Dystrophin; D016376:Oligonucleotides, Antisense