The potential of gene therapy for the treatment of disc degeneration.
Research in biologic methods of treating disc degeneration is still in its infancy. Many different strategies are being evaluated, but the gene therapy strategy stands out because of its potential for long-term efficacy. Choosing the correct gene for use in gene therapy is critically important. Of the many different classes of potentially therapeutic genes, the regulatory genes hold the most promise. Of the different gene therapy delivery methods, the most work has been performed with viral vectors, either ex vivo or in vivo. Current research now is turning toward in vivo experiments in rabbits. Efficacy and safety will be demonstrated first with smaller animal models. Beyond that, nonhuman primate experiments demonstrating efficacy and safety will be the penultimate step before initiation of human studies.
Yoon
S Tim
ST
Department of Orthopaedics, Veterans' Administration Medical Center, Decatur, GA 30033, USA. tim_yoon@emoryhealthcare.org
D048868:Adaptor Proteins, Signal Transducing; D055396:Bone Morphogenetic Protein 2; D055419:Bone Morphogenetic Protein 7; D019485:Bone Morphogenetic Proteins / Q000502:physiology; D002352:Carrier Proteins / Q000502:physiology; D002454:Cell Differentiation / Q000502:physiology; D003094:Collagen / Q000032:analysis; D003598:Cytoskeletal Proteins; D015316:Genetic Therapy; D006801:Humans; D007403:Intervertebral Disc / Q000737:chemistry / Q000473:pathology* / Q000503:physiopathology; D047908:Intracellular Signaling Peptides and Proteins; D060588:LIM Domain Proteins; D014161:Transduction, Genetic; D016212:Transforming Growth Factor beta / Q000502:physiology; D016335:Zinc Fingers / Q000502:physiology
D048868:Adaptor Proteins, Signal Transducing; C525718:BMP2 protein, human; C525819:BMP7 protein, human; D055396:Bone Morphogenetic Protein 2; D055419:Bone Morphogenetic Protein 7; D019485:Bone Morphogenetic Proteins; D002352:Carrier Proteins; D003598:Cytoskeletal Proteins; D047908:Intracellular Signaling Peptides and Proteins; D060588:LIM Domain Proteins; C484380:PDLIM7 protein, human; D016212:Transforming Growth Factor beta; D003094:Collagen