Gene therapy approaches for disc regeneration

One of the first articles describing the use of gene therapy was published in Science in 1972. In that paper, Friedmann and Roblin described how they induced mammalian cells to express bacterial and viral DNA, demonstrating the possibility that this method could later be used to promote therapies for treatment of human genetic diseases (Friedmann and Roblin 1972). Today, scientists are exploring the therapeutic use of gene therapy for the treatment of numerous pathological conditions including Parkinson disease (Yasuda et al. 2011), glioma (Lee et al. 2011), X-linked severe combined immunodeficiency (SCID-X) (Huston et al. 2011), diabetes (Rowzee et al. 2011), bone fractures (Sheyn et al. 2008; Kimelman-Bleich et al. 2011), and heart failure (Muona et al. 2012; Pleger et al. 2011). During the last two decades, efforts have been made to enhance the safety and efficiency of gene therapy. Despite numerous advances, the immune response, which led to the death of a patient in one clinical trial (Somia and Verma 2000), and insertional mutagenesis, which triggered the development of leukemia in several patients (Hacein-Bey-Abina et al. 2003; Cavazzana-Calvo et al. 2004), continue to pose a major barrier to the clinical use of gene therapy. Fortunately, improvements are constantly being made in protocols as well as in the safety features of some vectors, such as exogenous control of gene expression or the use of nonviral gene delivery methods (Herzog et al. 2010). Hundreds of clinical trials involving gene delivery are currently being conducted in the USA for the treatment of various diseases, ranging from cystic fibrosis to HIV (Flotte 2007).