Orthopedic Gene Therapy in 2008

被引:54
作者
Evans, Christopher H. [1 ]
Ghivizzani, Steven C. [2 ]
Robbins, Paul D. [3 ]
机构
[1] Harvard Univ, Sch Med, Beth Israel Deaconess Med Ctr, Ctr Mol Orthopaed, Boston, MA 02215 USA
[2] Univ Florida, Coll Med, Dept Orthopaed & Rehabil, Gene Therapy Lab, Gainesville, FL USA
[3] Univ Pittsburgh, Sch Med, Dept Microbiol & Mol Genet, Pittsburgh, PA USA
基金
美国国家卫生研究院;
关键词
MESENCHYMAL STEM-CELLS; BONE MORPHOGENETIC PROTEIN-2; ARTICULAR-CARTILAGE DEFECTS; ANTERIOR CRUCIATE LIGAMENT; GROWTH-FACTOR GENE; HUMAN INTERVERTEBRAL DISC; GENETICALLY-MODIFIED CHONDROCYTES; LIM MINERALIZATION PROTEIN-3; ADENOASSOCIATED VIRUS VECTOR; RECEPTOR-ANTAGONIST PROTEIN;
D O I
10.1038/mt.2008.265
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Orthopedic disorders, although rarely fatal, are the leading cause of morbidity and impose a huge socioeconomic burden. their prevalence will increase dramatically as populations age and gain weight. Many orthopedic conditions are difficult to treat by conventional means; however, they are good candidates for gene therapy. clinical trials have already been initiated for arthritis and the aseptic loosening of prosthetic joints, and the development of bone-healing applications is at an advanced, preclinical stage. other potential uses include the treatment of Mendelian diseases and orthopedic tumors, as well as the repair and regeneration of cartilage, ligaments, and tendons. Many of these goals should be achievable with existing technologies. the main barriers to clinical application are funding and regulatory issues, which in turn reflect major safety concerns and the opinion, in some quarters, that gene therapy should not be applied to nonlethal, nongenetic diseases. For some indications, advances in nongenetic treatments have also diminished enthusiasm. nevertheless, the preclinical and early clinical data are impressive and provide considerable optimism that gene therapy will provide straightforward, effective solutions to the clinical management of several common debilitating disorders that are otherwise difficult and expensive to treat.
引用
收藏
页码:231 / 244
页数:14
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