ABSTRACT
The ultimate goal is to replace a defective or missing gene with the normal copy in the patient's own tissues where the genes are normally expressed. The therapeutic outcome is achieved by the gene products delivered in vivo. Hence, the basis for somatic gene therapy has expanded to include both genetic and multifactorial disorders that can be treated by the delivery of recombinant gene products from genetically-modified host cells. Another primary requirement for recombinant gene products to be delivered with this microcapsule technology is that it must be a secretory gene product, a requirement not always met by the gene of interest. In summary, the delivery of recombinant gene products from genetically modified allogeneic cell lines has been proven feasible in vitro and in vivo. The clinical efficacy of this strategy to treat genetic disease has also been demonstrated in the Snell dwarf mice.
