REGENERATION OF PANCREATIC ISLETS AND REVERSAL OF DIABETES BY ISLET TRANSCRIPTION FACTOR GENES DELIVERED IN VIVO.

摘要

The present invention includes compositions and methods for regenerating glucose-responsive cells by ultrasound-targeted microbubble destruction in the pancreas, wherein the composition comprises a pre-assembled liposome-nucleic acid complex in contact with within and about a microbubble, wherein the pre-assembled liposome-nucleic acid complex comprises a NeuroD gene under the control of the promoter, wherein disruption of the microbubble in the pancreas at a target site delivers the nucleic acid into pancreas cells at the location of the ultrasound disruption, wherein cells that incorporate the nucleic acid express insulin in response to high blood glucose levels.