Vectors in gene therapy: Benefit for glioblastoma patients

Glioblastoma (GBM) is one of the most aggressive and lethal adult malignancies with less than 5% five years of survival average. Conventional treatment of tumor resection followed by radiation or chemotherapy is not effective due to the development of resistance. Therefore, there is an urgent need to develop new therapeutics to improve patient outcomes, and gene therapy is one of the options being considered. Although development of a novel effective stand-alone therapy is acceptable, therapeutics that improve patient’s response to existing treatments, both DNA damage inducing agents, are more practical. While the concept of gene therapy gave hope to potentially cure several diseases, some early failures grew skepticism. However, with notable steady advance, gene therapy is emerging as a formidable treatment option for diverse diseases, including GBM. Gene therapy mainly depends on viral and nonviral vectors to deliver a gene of interest into target cells. Vector-based gene therapy faces a challenge in the area, including but not limited to immune response, specificity, limited cargo size, and inefficient delivery. Despite the challenges, currently, there are numerous preclinical and clinical studies underway using nonviral and viral vectors. The advancement in gene therapy has created a renewed promise to benefit several hard to treat diseases, including GBM. In this chapter, the type of commonly used viral and nonviral vectors, as well as benefits for GBM, is discussed.