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Reversal of Diabetes in Pigs Using Liver-directed Gene Therapy

Project Member(s): Simpson, A., O'Brien, B.

Funding or Partner Organisation: National Health & Medical Research Council (NHMRC Project Grants)

Start year: 2008

Summary: Type I diabetes mellitus is caused by the autoimmune destruction of the beta cells of the pancreas that secrete insulin. We have shown that we can cure diabetes in spontaneuously diabetic mice by delivery of the insulin gene to the liver using a non-pathogenic viral delivery system. The study aims to repeat this work in pigs which have similiar physiology to humans. If successful this would be proof -of-principle that we could theoretically control blood glucose levels in humans.


Ren, B, La, QT, O'Brien, BA, Nassif, NT, Tan, Y, Gerace, D, Martiniello-Wilks, R, Torpy, F, Dane, AP, Alexander, IE & Simpson, AM 1970, 'EXPRESSION OF HUMAN PANCREATIC TRANSCRIPTION FACTORS IN THE LIVERS OF FRG MICE', JOURNAL OF GENE MEDICINE, Joint 10th Annual Scientific Meeting of the Australian-Gene-and-Cell-Therapy-Society (AGCTS) and Australasian-Society-for-Stem-Cell-Research (ASSCR), WILEY, Univ Technol Sydney, Sydney, AUSTRALIA.

Gerace, D, Martiniello-Wilks, R, Nassif, NT, Lal, S, Steptoe, R & Simpson, AM 2017, 'CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: a path to clinical success?', Stem Cell Research & Therapy, vol. 8, no. 1.
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Gerace, D, Martiniello-Wilks, R, O'Brien, BA & Simpson, AM 2015, 'The use of beta-cell transcription factors in engineering artificial beta cells from non-pancreatic tissue', GENE THERAPY, vol. 22, no. 1, pp. 1-8.
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Simpson, A, Gerace, D & Martiniello-Wilks, R 2015, 'Diabetes reversal via gene transfer: building on successes in animal models', Research and Reports in Endocrine Disorders, vol. 5, pp. 15-15.
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FOR Codes: Gene Therapy, Endocrine organs and diseases (incl. diabetes), Diabetes, Gene and Molecular Therapy, Gene and molecular therapy , Clinical health