Gene transfer to somatic tissues using retroviral vectors

Retroviral vectors have successfully been used for stable gene transfer in a number of in vivo situation. Before this approach can reallistically be applied in the treatment of human genetic diseases, the correction of pathological symptoms have to be documented in an animal model. In this perspective, we have chosen to use a strain of mice carrying the gusmps mutation, where homozygotes are deficient for beta-glucuronidase and develop a mucopolysaccharidosis. Two methods which result in the stable introduction of the human beta-glucuronidase cDNA into skin fibroblasts or hematopoietic stem cells are presented. A third method, whereby genes can be stably transferred to the liver is also discussed.