Pancreas-directed AAV8 -hSPINK1 gene therapy safely and effectively protects against pancreatitis in mice

OBJECTIVE: ) for pancreatitis therapy in mice. DESIGN: c.194+2T>C mouse models). RESULTS: before disease onset significantly alleviated the severity of pancreatitis, reduced the progression of fibrosis, decreased the levels of apoptosis and autophagy in the pancreas and accelerated the pancreatitis recovery process. CONCLUSION: safely targets the pancreas with high transduction efficiency and effectively ameliorates pancreatitis phenotypes in mice. This approach is promising for the prevention and treatment of CP.