Towards Stem Cell Treatment for Duchenne Muscular Dystrophy-Related Cardiomyopathy

Duchenne muscular dystrophy (DMD) is a progressive muscular degenerative disease affecting 1 in 3,500 boys. Cardiomyopathy is observed in 95% of patients aged 18 and older, accounting for 30% of deaths. While innovative treatments have been employed to curb symptoms in skeletal muscle, few show success in the heart, and no cure for DMD exists. Recently, regenerative therapy using cardiac stem cells (CSCs) has shown very promising results, but efficacy in DMD remains undetermined. This dissertation sought to provide preliminary insight into the effects of murine Sca-1+ CSC therapy. Three-dimensional echocardiography was validated and used to gauge cardiomyopathy in the mdx:utrn-/- mouse model. CSCs were isolated from healthy mice and displayed differentiation potential. They were implanted into mdx:utrn-/- mice under ultrasound guidance, and showed preliminary trends towards functional improvement. These results support the notion that stem cells may be an excellent avenue of therapy that should be further investigated