Human Embryonic Stem Cells in the Treatment of Patients With Duchenne Muscular Dystrophy: A Case Series

Abstract

Duchenne muscular dystrophy (DMD), an X-linked disorder, occurs due to mutation, deletion or duplication of the DMD gene which encodes the protein dystropin. It is estimated to affect 1 in 3,300 boys. The disease progression leads to muscles necrosis. In cases discussed in this article, five patients with DMD were treated with human embryonic stem cells (hESCs). All patients showed stability and mild improvement clinically like the ability to walk, stand, and sit, improved breathing capacity, increased body weight, improved hand functions and improved muscle strength. A remarkable reduction occurred in the creatine phosphokinase (CPK) levels of all the five patients without further deterioration. In conclusion, hESCs might become an effective therapeutic option for DMD in the future. More clinical data are needed to gather evidences supporting the use of hESC in the treatment of DMD.




J Neurol Res. 2015;5(1-2):186-191
doi: http://dx.doi.org/10.14740/jnr326w