Therapeutic strategies for the inherited neuropathies.
More than 30 genetic causes have been identified for the inherited neuropathies collectively referred to as Charcot-Marie-Tooth (CMT) disease. Previous therapies for CMT were limited to traditional approaches such as rehabilitation medicine, ambulation aids, and pain management. Identification of the genes causing CMT has led to improved genetic counseling and assistance in family planning. Identification of these genes is beginning to delineate common molecular pathways in multiple forms of CMT that can be exploited in future molecular therapies. Scientifically based clinical trials for CMT are currently being implemented. Techniques of gene therapy are advancing to the point that they may become feasible options for patients with CMT and other neurodegenerative diseases.
Shy
Michael E
ME
Department of Neurology and Center for Molecular Medicine and Genetics, Wayne State University School of Medicine, Detroit MI 48201, USA. m.shy@wayne.edu
D000818:Animals; D000975:Antioxidants / Q000627:therapeutic use; D001205:Ascorbic Acid / Q000627:therapeutic use; D001370:Axonal Transport / Q000502:physiology; D002462:Cell Membrane / Q000378:metabolism; D002607:Charcot-Marie-Tooth Disease / Q000503:physiopathology / Q000628:therapy*; D004721:Endoplasmic Reticulum / Q000378:metabolism; D018628:Gene Dosage; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D036341:Intercellular Signaling Peptides and Proteins / Q000627:therapeutic use; D008928:Mitochondria / Q000378:metabolism; D009186:Myelin Sheath / Q000378:metabolism / Q000473:pathology / Q000648:ultrastructure; D020933:Neurotrophin 3 / Q000627:therapeutic use; D010525:Peripheral Nerves / Q000378:metabolism / Q000503:physiopathology / Q000648:ultrastructure; D011374:Progesterone / Q000037:antagonists & inhibitors / Q000627:therapeutic use; D017510:Protein Folding; D012313:RNA / Q000378:metabolism; D012583:Schwann Cells
D000975:Antioxidants; D036341:Intercellular Signaling Peptides and Proteins; D020933:Neurotrophin 3; D011374:Progesterone; D012313:RNA; D001205:Ascorbic Acid