Growth Hormone Gene Therapy Using Encapsulated Myoblasts

摘要: Increasing numbers of gene therapy protocols are being accepted for human clinical trials both somatic and Mendelian genetic disorders. For monogenic diseases, the goal is to provide a normal level previously missing product in vivo (Morgan Anderson 1993, Morsy et al 1993). However, these products, which subject intricate metabolic control, treatment with has not yet progressed stage, either because our knowledge about regulatory control question limited, or technical capability reproduce pathway available. Hence, although coding insulin was one first genes cloned, diabetes still an unrealized goal. Secretion tightly controlled through interaction several pathways, its unregulated release by implanting insulin-secreting recombinant cells could lead severe hypoglycemia death (Selden 1987).