Transformation rate may be higher but rate of stable integrants still low. This complex has been linked to asialoglycoprotein which binds to hepatocytes and is internalized by receptor mediated endocytosis - cellular targetting. Gene gun fires DNA coated microscopic particles through cell walls and membranes. Direct injection into muscle cells - syncitium. Target Cells for ex vivo Transfer Hematopoietic stem cells - marrow or, recently, cord blood.
Precursor raises possibility of life long cure.
Appropriate for hematologic and immunologic defects. Small fraction of total cells. Methods being derived for enrichment. Embryonic stem cells in mice are a useful although not exactly equivalent model. Hepatocytes - liver lobe removal and culture possible with reinfusion into portal circulation.