Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by a progressive degeneration of muscles, and it is the most common cause of muscular dystrophy. Genetic mutation in dystrophin DMD gene have been reported in patients affected by DMD. Dystrophin is a cytoskeletal protein, and it is indispensable for the correct formation of the dystrophin-associated glycoprotein complexes (DGC) in muscle stem cells, and the sarcolemma of skeletal muscle, linking the muscle fiber sarcomere to the extracellular matrix in the skeletal muscle. Defects in dystrophin protein function result in abnormal muscle stem cell division during the muscle regeneration, and vulnerable muscle fibers due to mechanical stress during the muscle contraction. Through the identification of the genetic causes, novel treatments for DMD have been developed for more than thirty years, and translational research have produced several clinically relevant results, which will be potential therapeutic ways for DMD.
This Research Topic aims at highlighting current advances on therapeutic approaches for DMD, including stem cell therapy, gene therapy and other methods.
All topic editors declare no conflict of interest.
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by a progressive degeneration of muscles, and it is the most common cause of muscular dystrophy. Genetic mutation in dystrophin DMD gene have been reported in patients affected by DMD. Dystrophin is a cytoskeletal protein, and it is indispensable for the correct formation of the dystrophin-associated glycoprotein complexes (DGC) in muscle stem cells, and the sarcolemma of skeletal muscle, linking the muscle fiber sarcomere to the extracellular matrix in the skeletal muscle. Defects in dystrophin protein function result in abnormal muscle stem cell division during the muscle regeneration, and vulnerable muscle fibers due to mechanical stress during the muscle contraction. Through the identification of the genetic causes, novel treatments for DMD have been developed for more than thirty years, and translational research have produced several clinically relevant results, which will be potential therapeutic ways for DMD.
This Research Topic aims at highlighting current advances on therapeutic approaches for DMD, including stem cell therapy, gene therapy and other methods.
All topic editors declare no conflict of interest.
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
