Selective Strike: New Gene Therapy Destroys Kaposi’s Sarcoma Cells, Spares Healthy Tissue

UC Davis researchers have developed a novel “Trojan horse” gene therapy that specifically kills cancer cells infected with Kaposi’s sarcoma–associated herpesvirus (KSHV), while sparing healthy uninfected cells.

In preclinical experiments using human cell cultures and mouse models, the therapy halted tumor growth without obvious side effects. The approach leverages an adeno‑associated virus (AAV) vector engineered to activate only in cells expressing the viral latency protein LANA, which marks KSHV‑infected cancer cells.

Once inside a KSHV‑positive cell, the vector supplies a gene for a modified thymidine kinase enzyme. When patients (or in models, mice) are treated with the antiviral drug ganciclovir, that enzyme converts the drug selectively into a toxic compound that kills the infected cancer cell. In laboratory tests, uninfected cells were largely unaffected.

Interestingly, the team also found that certain anti‑cancer agents that re-activate viral gene expression actually enhance the therapy’s effectiveness by increasing the LANA activity, thereby amplifying the system’s targeting.

KSHV is implicated in Kaposi’s sarcoma and several rare lymphomas. Conventional therapies for such virus‑associated cancers often carry substantial toxicity and limited specificity. The new strategy offers a more precise assault on virus‑bearing tumor cells.

Though still in early stages, the researchers hope to move toward human trials. Their long-term goal is to transform how virus‑driven cancers are treated by turning the virus’s own biology into a therapeutic vulnerability.