AAV vectors for cancer gene therapy

Current cancer therapies, including cytotoxic agents, molecularly targeted drugs, and immune-based treatments, are often constrained by therapeutic resistance, systemic toxicity, and inconsistent clinical efficacy. The adeno-associated virus (AAV) vector-based therapy emerges as a promising strategy to address these limitations, owing to its favorable safety profile and high degree of engineerability. AAV-based gene therapy development is expanding beyond the treatment of rare monogenic disorders to encompass chronic diseases and cancer conditions associated with a high disease burden, driven not only by advances in vector technology but also by strategic considerations such as development costs, clinical needs, and commercial viability. Although challenges such as pre-existing host immunity and limited transgene packaging capacity remain, recent advances in biotechnology are actively mitigating these barriers. This review highlights recent progress in AAV-based gene therapy for cancer challenges and discusses future directions for the implementation of AAV vectors as a next-generation therapeutic modality in oncology.