Improving Safety of Gene Therapy

Title: Improving Safety of Gene Therapy

Volume: 3 Issue: 1

Author(s): Seppo Yla-Herttuala, Jani K. Raty, Hanna P. Lesch and Thomas Wirth

Affiliation:

Keywords: Gene therapy, safety, AAV, adenovirus, retrovirus

Abstract: Gene therapy has progressed from early clinical trials to first commercial gene therapy drugs. While there is a long history with the side-effects and adverse effects of pharmaceutical drugs, drugs based on gene delivery have presented new challenges for researchers, clinicians and regulatory authorities. On the path from early pre-clinical research to final commercial products, gene therapy tools and production methods have undergone tremendous changes to improve safety and efficacy. Deletion of adenovirus replication genes E1 and E3 has progressed to gutless adenoviruses with all viral genes removed; similarly evolution of lentiviral vectors has progressed from first generation viruses to safer third generation self-inactivating vectors. Improved chromatographic methods have eased the purification of viruses and delivery reservoirs, such as collagen or silicon collars for cardiovascular gene transfer have decreased systemic leakage of viruses; together with tissue-specific promoters and imaging of the biodistribution of viral particles, gene therapy specificity and safety can be improved even further. This review will introduce gene delivery vectors used in gene therapy and highlight key approaches used to improve their safety.

Cite this article as:

Yla-Herttuala Seppo, Raty K. Jani, Lesch P. Hanna and Wirth Thomas, Improving Safety of Gene Therapy, Current Drug Safety 2008; 3 (1) . https://dx.doi.org/10.2174/157488608783333925