Gene therapy is the introduction or removal of genetic material or modification of gene expression to alter the biological function of an individual’s genetic code with the objective of achieving a therapeutic benefit. Gene therapies can be delivered either by transporting the gene directly into the body (in vivo)17 or by modifying cells and returning them into the body (ex vivo).18,19 Gene therapy approaches include ‘knocking out’ or inactivating, a mutated gene that is functioning improperly; introducing new genes into the cells or replacing a mutated gene that causes disease with a healthy gene. Gene replacement therapy utilizes a delivery vehicle, a viral vector carrying the transgene to supply the healthy gene to the cell. This allows for restoration of function of the defected gene and production of the protein of interest.
Gene editing is a process of making precise alterations to an organism's DNA, which can be used to add, remove or alter genes at specific sites in the genome. While techniques to modify DNA have existed for several decades, new methods have made genome editing faster, cheaper and more efficient.
- American Society of Gene and Cell Therapy. Gene and cell therapy FAQs, 2018. Available at: https://www.asgct.org/education/gene-and-cell-therapy-faqs. [Accessed March 25, 2023].
- American Society of Gene & Cell Therapy. Gene and cell therapy FAQs, 2018. Available at: https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs. [Accessed March 25, 2023].
- Keeler AM, ElMallah MK, Flotte TR. Gene therapy 2017: progress and future directions. Clin Transl Sci. 2017:10(4):242-248.
- Sung, Y., Kim, S. Recent advances in the development of gene delivery systems. Biomater Res 23, 8 (2019). https://doi.org/10.1186/s40824-019-0156-z.
- Bulcha, J.T., Wang, Y., Ma, H. et al. Viral vector platforms within the gene therapy landscape. Sig Transduct Target Ther 6, 53 (2021). https://doi.org/10.1038/s41392-021-00487-6.
- American Society of Gene and Cell Therapy. Gene and cell therapy FAQs. 2018. Available at: https://www.asgct.org/education/gene-and-cell-therapy-faqs. [Accessed: March 25, 2023].