[Molecular therapy of muscular dystrophy].
Muscular dystrophy is a nosology for a group of hereditary muscle disorders characterized by progressive wasting and weakness of skeletal muscle, where degeneration of muscle fibers is detected by pathological examination. Since the causative gene of Duchenne muscular dystrophy (DMD), the most severe and abundant form of muscular dystrophy, the DMD gene, and its product dystrophin was isolated by positional cloning by Dr. Kunkel and his colleagues, the studies on molecular pathologies of muscular dystrophy has been extensively developed. The current therapeutic approaches of muscular dystrophy, such as DMD involves pharmacological suppression of the inflammatory and immure responses, which usually provides only modest and temporary beneficial effects. Future approaches depend on cell and gene therapy technology and will require different strategies, none of which are currently ready to enter clinical practice. These approaches involve the efficient, non-antigenic gene transfer for in vivo gene therapy, pharmacological upregulation of the synthesis of utrophin, a related protein that compensates for the loss of dystrophin, and myogenic stem cell transplantation. These approaches could be integrated each other and called as molecular therapy.
Takeda
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D000818:Animals; D003598:Cytoskeletal Proteins / Q000378:metabolism; D016189:Dystrophin / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D018380:Hematopoietic Stem Cell Transplantation; D008565:Membrane Proteins / Q000378:metabolism; D009136:Muscular Dystrophies / Q000235:genetics* / Q000628:therapy*; D049411:Utrophin
D003598:Cytoskeletal Proteins; D016189:Dystrophin; D008565:Membrane Proteins; D049411:Utrophin