Orthopedic gene therapy in 2008

Christopher H Evans, Steven C. Ghivizzani, Paul D. Robbins

Research output: Contribution to journalArticle

61 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)231-244
Number of pages14
JournalMolecular Therapy
Volume17
Issue number2
DOIs
StatePublished - 2009
Externally publishedYes

Fingerprint

Genetic Therapy
Orthopedics
Musculoskeletal Diseases
Bone Development
Ligaments
Tendons
Weight Gain
Arthritis
Cartilage
Regeneration
Joints
Clinical Trials
Technology
Morbidity
Safety
Therapeutics
Population
Neoplasms
Optimism

ASJC Scopus subject areas

  • Genetics
  • Molecular Biology
  • Molecular Medicine
  • Medicine(all)
  • Drug Discovery
  • Pharmacology

Cite this

Evans, C. H., Ghivizzani, S. C., & Robbins, P. D. (2009). Orthopedic gene therapy in 2008. Molecular Therapy, 17(2), 231-244. https://doi.org/10.1038/mt.2008.265

Orthopedic gene therapy in 2008. / Evans, Christopher H; Ghivizzani, Steven C.; Robbins, Paul D.

In: Molecular Therapy, Vol. 17, No. 2, 2009, p. 231-244.

Research output: Contribution to journalArticle

Evans, CH, Ghivizzani, SC & Robbins, PD 2009, 'Orthopedic gene therapy in 2008', Molecular Therapy, vol. 17, no. 2, pp. 231-244. https://doi.org/10.1038/mt.2008.265
Evans, Christopher H ; Ghivizzani, Steven C. ; Robbins, Paul D. / Orthopedic gene therapy in 2008. In: Molecular Therapy. 2009 ; Vol. 17, No. 2. pp. 231-244.
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