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Gene therapy in orthopaedic surgery: the current status.
Mia Jüllig1, Wei V Zhang, N Susan Stott
1Orthopaedic Research Laboratory, Division of Surgery, Faculty of Medicine and Health Sciences, University of Auckland, Auckland, New Zealand.
ANZ Journal of Surgery
|January 17, 2004
Summary
Gene therapy has advanced significantly since 1991, with numerous trials and patient involvements. However, limited published data and safety concerns necessitate further evaluation, especially for orthopaedic applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Medical Research
Background:
- Gene therapy trials began in 1991, showing successful gene transfer and expression in early studies.
- Over 400 clinical protocols involving more than 4000 patients have been approved.
- Publication of trial results remains limited, with only 80 reports by 2002.
Purpose of the Study:
- To review the current status of gene therapy in orthopaedic diseases.
- To identify areas requiring further progress in the field.
- To address safety concerns and the clinical role of gene therapy.
Main Methods:
- Review of published gene therapy trial results.
- Analysis of safety data from clinical protocols.
- Focus on gene therapy applications in orthopaedics.
Main Results:
- Majority of trials are Phase I/II, focusing on safety.
- Safety concerns, including patient death and leukemia development, have prompted re-evaluation.
- Transitory high levels of exogenous gene expression achieved in many studies.
Conclusions:
- Gene therapy shows promise but faces challenges in widespread clinical application.
- Further research is needed to ensure safety and efficacy, particularly for orthopaedic conditions.
- The role of gene therapy in clinical practice requires ongoing assessment.