Method of delivering vectors to pancreas and lungs by cannulating the aorta

摘要

The present invention relates to a safe and effective way to deliver and express therapeutic compositions (e.g., transgenes) to the pancreas and lungs of a mammal.

主权项

1. A method of administering a therapeutic composition to a mammal in need thereof comprising:
(a) inserting a catheter into an umbilical or femoral artery in the mammal, (b) advancing the catheter to the aorta (celiac or superior mesenteric artery), and (c) delivering a therapeutic composition through the catheter to the celiac or superior mesenteric artery in the mammal,
wherein the therapeutic composition is an adeno-associated viral-9 (AAV9) vector encoding a nucleic acid encoding a therapeutic substance, and wherein the therapeutic substance is expressed in pancreatic duct epithelial cells or pancreatic polypeptide secreting cells of islets of the mammal, and wherein insulin, glucagon, somatostatin-secreting cells and acinar cells are not transduced in the mammal.