Duchenne Muscular Dystrophy

This volume assesses research, in humans and in animals, on myoblast transfer and gene therapy for Duchenne muscular dystrophy (DMD). The contributors describe and compare two different animal models used in current experiments on DMD therapy: the xmd dog, which exhibits skeletal muscular dystrophy similar to human DMD, and the mdx mouse, which shares the genetic dystrophin defect underlying the disease, but does not develop severe progressive skeletal myopathy. Noted experts analyze studies on myoblast transfer in DMD patients and animals, explore new strategies for direct gene transfer in DMD, and discuss the relative feasibility of donor myoblast transfer, direct gene transfer, and patient myoblast-mediated gene transfer.
 
This volume assesses research, in humans and in animals, on myoblast transfer and gene therapy for Duchenne muscular dystrophy (DMD). The contributors describe and compare two different animal models used in current experiments on DMD therapy: the xmd dog, which exhibits skeletal muscular dystrophy similar to human DMD, and the mdx mouse, which shares the genetic dystrophin defect underlying the disease, but does not develop severe progressive skeletal myopathy. Noted experts analyze studies on myoblast transfer in DMD patients and animals, explore new strategies for direct gene transfer in DMD, and discuss the relative feasibility of donor myoblast transfer, direct gene transfer, and patient myoblast-mediated gene transfer.