Antisense-based therapy for the treatment of spinal muscular atrophy

作者:Rigo Frank; Hua Yimin; Krainer Adrian R; Bennett C Frank*
来源:The Journal of Cell Biology, 2012, 199(1): 21-25.
DOI:10.1083/jcb.201207087

摘要

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.

  • 出版日期2012-10-1