Antisense Therapy in Neurology

Journal of Personalized Medicine

Joshua J.A. Lee¹ 

[1] Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada; E-Mail:

关键词: Duchenne muscular dystrophy (DMD);    Fukuyama congenital muscular dystrophy (FCMD);    myotonic dystrophy (DM);    spinal muscular atrophy (SMA);    Huntington’s disease (HD);    amyotrophic lateral sclerosis (ALS);    limb-girdle muscular dystrophy 2B (LGMD2B);    Miyoshi myopathy (MM);    distal myopathy with anterior tibial onset (DMAT);    antisense therapy;   

DOI  :  10.3390/jpm3030144

来源: mdpi

【 摘 要 】

Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders. Previous and ongoing pre-clinical and clinical trials have provided encouraging early results. Spinal muscular atrophy (SMA), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Fukuyama congenital muscular dystrophy (FCMD), dysferlinopathy (including limb-girdle muscular dystrophy 2B; LGMD2B, Miyoshi myopathy; MM, and distal myopathy with anterior tibial onset; DMAT), and myotonic dystrophy (DM) are all reported to be promising targets for antisense therapy. This paper focuses on the current progress of antisense therapies in neurology.

【 授权许可】

CC BY   
© 2013 by the authors; licensee MDPI, Basel, Switzerland.