%0 Journal Article
%T Antisense Therapy in Neurology
%A Joshua J. A. Lee
%A Toshifumi Yokota
%J Journal of Personalized Medicine
%D 2013
%I MDPI AG
%R 10.3390/jpm3030144
%X 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), HuntingtonĄ¯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.
%K Duchenne muscular dystrophy (DMD)
%K Fukuyama congenital muscular dystrophy (FCMD)
%K myotonic dystrophy (DM)
%K spinal muscular atrophy (SMA)
%K HuntingtonĄ¯s disease (HD)
%K amyotrophic lateral sclerosis (ALS)
%K limb-girdle muscular dystrophy 2B (LGMD2B)
%K Miyoshi myopathy (MM)
%K distal myopathy with anterior tibial onset (DMAT)
%K antisense therapy
%U http://www.mdpi.com/2075-4426/3/3/144