Cell targeting by murine retroviral vectors.

Gene therapy involves the transfer of new genetic material to cells of individuals with the aim of conferring a therapeutical benefit. Theoretically, gene therapy can be used to treat a variety of life-threatening disorders, such as inherited genetic diseases, cancer, chronic viral infections as well as a number of other severe diseases which are not treatable at present. To this aim, both efficient gene delivery techniques and controlled gene expression systems are required. Engineered murine retroviruses are the most widely used vectors for stable clinical gene transfer because of their ability to integrate--along with the transgenes they are engineered to carry--into the genome of infected cells. However, this technology still suffers from a number of drawbacks and limitations. Particularly, the ability to target cells of therapeutic interest together with the controlled expression of transferred genes would improve both the efficiency and the safety of retroviral vectors. Such improvements would additionally allow the development of new animal models of human diseases as well as enable more fundamental investigations in the fields of oncogenesis and developmental biology.