Academic Journals Database
Disseminating quality controlled scientific knowledge

Antisense Therapy in Neurology

ADD TO MY LIST
 
Author(s): Joshua J. A. Lee | Toshifumi Yokota

Journal: Journal of Personalized Medicine
ISSN 2075-4426

Volume: 3;
Issue: 3;
Start page: 144;
Date: 2013;
Original page

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

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.

Tango Jona
Tangokurs Rapperswil-Jona

     Affiliate Program