关键词：医药；治疗技术；基因治疗；脊柱融合
摘 要：Surgery of the spine to fuse the vertebral bones is one of the most commonly performed operations with an estimated 350,000 Americans undergoing this surgery annually with estimated costs of $60 billion. Current procedures are highly invasive with limited success. The goal of this study is to develop a safe efficacious system for inducing spine fusion which will eliminate the need for invasive surgery. We have currently developed a cell based gene therapy system that can induce rapid bone formation at a targeted location which is independent of immune status of the model. This system relies on adenovirus transduced cells expressing bone morphogenetic protein 2 to induce bone formation leading to vertebral fusion after delivery into the paraspinous musculature. To prolong cell survival and insure cells are maintained at the target site, we have encapsulated them in a nondegradable hydrogel material. This provides additional safety by eliminating direct injection of the virus through cell delivery, and prevention of cell diffusion, through encapsulation. Here we provide preliminary data; demonstrating spine fusion using this system at 6 weeks after induction. This is the first step in demonstrating efficacy, a critical component of preclinical testing. Thus with validation of our hypothesis, this approach can now be developed as a safe and efficacious gene therapy system for spine fusion, thus circumventing the need for costly invasive surgery.