الفهرس | Only 14 pages are availabe for public view |
Abstract Gene Therapy is the treatment of a disease by expression of one or multiple specific genes in a cell or group of cells. The Production of the desired gene product corrects the genetic defect or alters cellular function. Our knowledge of gene expression and regulation plays a critical role in the design of gene therapy studies. Despite the difficulty in safety and efficiency of gene delivery, a few successful clinical studies have shown the feasibility of using gene therapy to cure fatal human diseases. Work in this field not only enhanced our understanding of the problems involved in gene therapy, but also will allow us to continue improving our methodology. Although the obstacles discussed here still impede our current progress in bringing clinical benefits of gene therapy to patients, all these problems can be solved theoretically. |