Copyright
©The Author(s) 2015.
World J Clin Pediatr. Nov 8, 2015; 4(4): 66-80
Published online Nov 8, 2015. doi: 10.5409/wjcp.v4.i4.66
Published online Nov 8, 2015. doi: 10.5409/wjcp.v4.i4.66
Figure 4 Novel therapies using RNA-based mechanisms to mediate RNA toxicity in a myotonic dystrophy type 1 cell.
Promising trials have shown various means and targets of RNA mediated therapy with the aim of reversing or modifying “spliceopathy” and normalising cellular splice protein levels and actions. MBNL: Musclebind-like protein.
- Citation: Ho G, Cardamone M, Farrar M. Congenital and childhood myotonic dystrophy: Current aspects of disease and future directions. World J Clin Pediatr 2015; 4(4): 66-80
- URL: https://www.wjgnet.com/2219-2808/full/v4/i4/66.htm
- DOI: https://dx.doi.org/10.5409/wjcp.v4.i4.66