title: Antisense-based therapy for the treatment of spinal muscular atrophy.
abstract: One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.
authors.affiliation: Isis Pharmaceuticals, Carlsbad, CA 92010, USA.
authors.forename: Frank
authors.initials: F
authors.lastname: Rigo
authors.forename: Yimin
authors.initials: Y
authors.lastname: Hua
authors.forename: Adrian R
authors.initials: AR
authors.lastname: Krainer
authors.forename: C Frank
authors.initials: CF
authors.lastname: Bennett
mesh_terms: D000818:Animals; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy* / Q000235:genetics; D016376:Oligonucleotides, Antisense / Q000494:pharmacology*; D012333:RNA, Messenger / Q000187:drug effects / Q000235:genetics; D055533:Survival of Motor Neuron 1 Protein / Q000037:antagonists & inhibitors / Q000235:genetics; D055540:Survival of Motor Neuron 2 Protein / Q000037:antagonists & inhibitors / Q000235:genetics
chemical_list: D016376:Oligonucleotides, Antisense; D012333:RNA, Messenger; C526471:SMN1 protein, human; C526477:SMN2 protein, human; D055533:Survival of Motor Neuron 1 Protein; D055540:Survival of Motor Neuron 2 Protein