Abstract
The potential for nonviral gene therapy to treat cancer has been limited by the difficulty of targeting therapeutic genes to tumors. In this issue of Molecular Therapy, Senzer et al.1 show evidence of specific tumor targeting as well as clinical efficacy in a phase I trial of a nonviral vector. This is the first phase I clinical trial to demonstrate tumor specificity and uptake by metastatic tumors of a systemically delivered liposomal nanoparticle. Importantly, the therapeutic nanoparticle elicited minimal side effects and the majority of patients demonstrated stable disease. This phase I trial has shown this delivery system to be a safe, stable, efficient, and tumor-specific drug delivery platform for systemic administration.
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