Aartsma-Rus Annemieke
Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Methods Mol Biol. 2012;867:97-116. doi: 10.1007/978-1-61779-767-5_7.
Antisense-mediated exon skipping to restore the disrupted dystrophin reading frame is currently in clinical trials for Duchenne muscular dystrophy. This chapter describes the rationale of this approach and gives an overview of in vitro and in vivo experiments with antisense oligonucleotides and antisense genes. Finally, an overview of clinical trials is given and outstanding questions and hurdles are discussed.
反义介导的外显子跳跃以恢复中断的抗肌萎缩蛋白读码框目前正在进行杜氏肌营养不良症的临床试验。本章描述了这种方法的原理，并概述了使用反义寡核苷酸和反义基因进行的体外和体内实验。最后，给出了临床试验的概述，并讨论了悬而未决的问题和障碍。
