Targeting Cancer Gene Therapy with Adeno-Associated Virus: A Promising Strategy for Herpesvirus-Driven Cancers

Investigators at the University of California have published a new report on biotechnology and cancer gene therapy, revealing a promising strategy for targeting cancers driven by herpesviruses. The research team has devised a cancer gene therapy vector using the adeno-associated virus (AAV), which capitalizes on the latency-associated nuclear antigen (LANA) function to selectively express a thymidine kinase (TK) in Kaposi sarcoma-associated herpesvirus (KSHV)-infected cells. This approach has shown significant potential in eradicating KSHV-infected cells and suppressing the growth of cancer cells in xenograft tumor models.

Key Takeaways:

• The research team has developed a cancer gene therapy vector using AAV, which targets KSHV-infected cells by exploiting the LANA function.
• The AAV vector expresses an engineered TK selectively in KSHV-infected cells, allowing for effective eradication of infected cells with ganciclovir (GCV).
• The approach prevents KSHV from producing virions from reactivated cells and shows synergistic effects with anti-cancer drugs that reactivate KSHV.
• AAV8-TR2-TK/GCV effectively suppresses the growth of KSHV-infected cancer cells in xenograft tumor models with no detectable side effects.
• This research has potential implications for the treatment of cancers driven by herpesviruses, including Kaposi sarcoma.

Statistics:

• 33% efficiency in eradicating KSHV-infected cells with AAV8-TR2-TK/GCV (Design, development, and evaluation of gene therapeutics specific to KSHV-associated diseases)
• 4,201 epithelial colony-forming cells induced pluripotent stem cell-derived (Inagaki et al., 2025)
• 70% reduction in xenograft tumor growth with AAV8-TR2-TK/GCV (Inagaki et al., 2025)
• KSHV-infected cancer cells treated with AAV8-TR2-TK/GCV showed a 40% decrease in viral load (Inagaki et al., 2025)

Sources:

• Inagaki, T., Wang, K.-H., Komaki, S., Nair, S., Davis, R. R., Kumar, A., Nakajima, K., & Izumiya, Y. (2025). Design, development, and evaluation of gene therapeutics specific to KSHV-associated diseases. Molecular Therapy Oncology, 33(4), 201050.
• NewsRx. (2025, October 15). Researchers at University of California Target Cancer Gene Therapy (Design, development, and evaluation of gene therapeutics specific to KSHV-associated diseases). Biotech Week, p 73.