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The application of liver gene therapy is being evaluated in two areas: single gene disorders of the liver, using the LEC rat model of Wilson’s disease as a model system; and the in vivo transdifferentiation of hepatocytes into pancreatic β cells for the treatment of type 1 diabetes.
Another important research area for the group is the development of peptide nanoparticles for the delivery of hydrophilic therapeutic moieties directly to cytolsol.
Clinical Sciences
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DESCRIPTION
Clinical Sciences’ major area of research is the development of safe and effective non-viral techniques for gene delivery to the liver, concentrating on two approaches: the hydrodynamic approach, and the use of synthetic peptide motifs to enable peptide/DNA particles to enter the cytosol and translocate to the nucleus. The application of liver gene therapy is being evaluated in two areas: single gene disorders of the liver, using the LEC rat model of Wilson’s disease as a model system; and the in vivo transdifferentiation of hepatocytes into pancreatic β cells for the treatment of type 1 diabetes.
Another important research area for the group is the development of peptide nanoparticles for the delivery of hydrophilic therapeutic moieties directly to cytolsol.
Associated research programmes
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CONTACTS FOR FURTHER INFORMATION
Professor John Fabre
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