Antisense oligonucleotides shed new light on the pathogenesis and treatment of spinal muscular atrophy.
Department of Virology, Beckman Research Institute, City of Hope National Medical Center, Duarte, California 91010, USA. jyee@coh.org
Jiing-Kuan
JK
Yee
Ren-Jang
RJ
Lin
D017398:Alternative Splicing; D000818:Animals; D015316:Genetic Therapy; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000235:genetics* / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D055533:Survival of Motor Neuron 1 Protein / Q000235:genetics
D016376:Oligonucleotides, Antisense; D055533:Survival of Motor Neuron 1 Protein