Development of engineered cells for implantation in gene therapy

Human gene therapy is based on the technology of genetic engineering of cells, either through ex vivo or in vivo methods of gene transfer. Many autologous cell types have been successfully modified to deliver recombinant gene products. An alternate form of gene therapy based on genetic modification of non-autologous cells is described. Protection within immuno-isolating devices would allow implantation of well-established recombinant cell lines in different allogeneic hosts, potentially offering a more cost-effective approach to gene therapy. Implantation with microencapsulated fibroblasts and myoblasts has resulted in successful recombinant product delivery in vivo. Correction of disease phenotypes in animal models of human genetic diseases has also been achieved. Cell types such as myoblasts which can differentiate terminally within the implantation device are particularly promising for the future development of this method of gene therapy.