Genome engineering and disease modeling via programmable nucleases for insulin gene therapy: Promises of CRISPR/Cas9 technology

Figure 4 Testing the therapeutic efficacy of the lentivirus vector carrying insulin gene (LentiINS). The chemotherapeutic agent Streptozotocin can be used to destroy endogenous pancreatic beta cells to induce an experimental animal model of diabetes. Insulin knockout (INS KO) pancreatic beta cells after transduction with the LentiINS can be transplanted under the kidney capsule of diabetic animals. Diabetic animals transplanted with the LentiINS-transduced INS KO pancreatic beta cells are expected to lower the blood glucose. On the other hand, hyperglycemia is anticipated in diabetic animals transplanted with INS KO pancreatic beta cells alone. STZ: Streptozotocin; KO: Knockout.