Cutting through the obstacles and resurrecting the promise of gene therapy

Gene therapy is a promising biomedical discipline that could potentially lead to new treatments and perhaps long-term curative effects for a plethora of diseases including hereditary disorders, cardiovascular and neurological diseases, cancer, diabetes and even infectious or autoimmune diseases. These diseases affect millions of people worldwide and the development of effective and safe gene-based drugs obviously represent a tremendous market potential. Convincing evidence continues to emerge from clinical trials demonstrating that gene therapy can be effective in patients suffering from a limited number of different diseases. Nevertheless, as with any emerging new biomedical discipline, gene therapy has also faced a number of setbacks, and there have been concerns regarding the safety of some gene delivery approaches, however, these hurdles are not insurmountable. Gene transfer technologies are improving rapidly and have led to the development of new and more efficacious gene delivery approaches with fewer side effects. The success of gene therapy is still highly dependent upon the continuous development of improved gene delivery technologies, the progress of which should hopefully and ultimately cure diseases that are refractory to current treatment paradigms.