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Review
. 2009 Feb;17(2):231-44.
doi: 10.1038/mt.2008.265. Epub 2008 Dec 9.

Orthopedic gene therapy in 2008

Christopher H Evans  1 , Steven C Ghivizzani, Paul D Robbins
Affiliations
Review

Orthopedic gene therapy in 2008

Christopher H Evans et al. Mol Ther. 2009 Feb.

Abstract

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.

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Figures

<b>Figure 1</b>
Figure 1
Categories of orthopaedic conditions potentially amenable to gene therapy. CACP, campodactyly-arthropathy-coax vera-pericarditis. From ref. 3 with permission.
<b>Figure 2</b>
Figure 2
Strategies for the gene therapy of arthritis. Modified from refs. 16 and 166, with permission.
<b>Figure 3</b>
Figure 3
Gene therapy strategies for tissue repair and regeneration. GAM, gene-activated matrix.
<b>Figure 4</b>
Figure 4
Progress in the development of gene therapies for various orthopedic applications. RA, rheumatoid arthritis; OA, osteoarthritis; As. Loose., aseptic loosening; Tend.-Lig., tendon and ligament; IDD, intervertebral disc degeneration; Osteoporo, osteoporosis; Mendelian, Mendelian diseases, excluding Gaucher.
See this image and copyright information in PMC

References

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