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• Yokota, T
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J. Pers. Med. 2013, 3(3), 144-176; doi:10.3390/jpm3030144

Review

Antisense Therapy in Neurology

Joshua J. A. Lee ¹ and Toshifumi Yokota ^1,2,*

¹ Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, 8812-112 St, Edmonton T6G 2H7, Canada ² The Friends of Garrett Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, 8812-112 St, Edmonton T6G 2H7, Canada

* Author to whom correspondence should be addressed.

Received: 27 May 2013; in revised form: 26 July 2013 / Accepted: 29 July 2013 / Published: 2 August 2013

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Abstract: 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.

Keywords: 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

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