Future Directions in Gene Therapy Research for Monogenic Diseases
Author Information
Author(s): Matthew H. Porteus, Jon P. Connelly, Shondra M. Pruett
Primary Institution: University of Texas Southwestern Medical Center
Hypothesis
How can gene therapy be improved to minimize risks such as insertional oncogenesis?
Conclusion
Gene therapy has shown promise in treating monogenic diseases, but significant challenges remain, particularly regarding safety and efficacy.
Supporting Evidence
- Gene therapy has been beneficial for some patients with severe combined immunodeficiency (SCID).
- Adverse events in gene therapy trials have highlighted the need for improved safety measures.
- Developing quantitative assays for risk and efficacy is essential for the future of gene therapy.
Takeaway
Gene therapy aims to fix diseases caused by single gene changes, but researchers need to find safer ways to do it to avoid problems like cancer.
Methodology
This review discusses recent gene therapy trials and identifies areas for future research, focusing on the risks and efficacy of gene therapy protocols.
Potential Biases
Potential bias in interpreting the safety and efficacy of gene therapy based on limited trial data.
Limitations
Current preclinical safety models are inadequate for assessing oncogenic risk due to insufficient integration event analysis and short observation periods.
Digital Object Identifier (DOI)
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