Diabetes reversal via gene transfer: building on successes in animal models

Gerace, D; Martiniello-Wilks, R; Simpson, AM

Diabetes reversal via gene transfer: building on successes in animal models

Gerace, D

Martiniello-Wilks, R

Simpson, AM

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Publisher:: Dove Medical Press
Publication Type:: Journal Article
Citation:: Research and Reports in Endocrine Disorders, 2015, 5 pp. 15 - 29
Issue Date:: 2015-01-29

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Field	Value	Language
dc.contributor.author	Gerace, D https://orcid.org/0000-0001-9154-0624	en_US
dc.contributor.author	Martiniello-Wilks, R https://orcid.org/0000-0002-0038-3430	en_US
dc.contributor.author	Simpson, AM https://orcid.org/0000-0002-2249-5097	en_US
dc.date.issued	2015-01-29	en_US
dc.identifier.citation	Research and Reports in Endocrine Disorders, 2015, 5 pp. 15 - 29	en_US
dc.identifier.issn	2230-2271	en_US
dc.identifier.uri	http://hdl.handle.net/10453/32759
dc.description.abstract	Type 1 diabetes (T1D) is caused by the autoimmune destruction of the insulin-producing pancreatic β-cells. People with T1D manage their hyperglycemia using daily insulin injections; however, this does not prevent the development of long-term diabetic complications such as retinopathy, nephropathy, neuropathy, and various macrovascular disorders. Currently, the only "cure" for T1D is pancreas transplantation or islet-cell transplantation; however, this is hampered by the limited number of donors and the requirement for life-long immunosuppression. As a result, the need for alternative therapies is vital. One of the strategies employed to correct T1D is the use of gene transfer to generate the production of an “artificial” β-cell that is capable of secreting insulin in response to fluctuating glucose concentrations that normally occurs in people without T1D. The treatment of many diseases using cell and gene therapy is generating significant attention in the T1D research community; however, for a cell therapy to enter clinical trials, success and safety must first be shown in an appropriate animal model. Animal models have been used in diabetes research for over a century, have improved our understanding of the pathophysiology of diabetes, and have led to the discovery of useful drugs for the treatment of the disease. Currently, the nonobese diabetic mouse is the animal model of choice for the study of T1D as it most closely reflects disease development in humans. The aim of this review is to evaluate the success of cell and gene therapy to reverse T1D in animal models for future clinical application.	en_US
dc.publisher	Dove Medical Press	en_US
dc.relation	http://purl.org/au-research/grants/nhmrc/352909
dc.relation	http://purl.org/au-research/grants/nhmrc/513100
dc.relation.ispartof	Research and Reports in Endocrine Disorders	en_US
dc.relation.isbasedon	10.2147/RRED.S51644	en_US
dc.title	Diabetes reversal via gene transfer: building on successes in animal models	en_US
dc.type	Journal Article
utslib.citation.volume	5	en_US
utslib.for	0604 Genetics	en_US
utslib.for	100401 Gene and Molecular Therapy	en_US
utslib.for	1103 Clinical Sciences	en_US
pubs.embargo.period	Not known	en_US
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Science
pubs.organisational-group	/University of Technology Sydney/Faculty of Science/School of Life Sciences
pubs.organisational-group	/University of Technology Sydney/Strength - CHT - Health Technologies
utslib.copyright.status	open_access
pubs.consider-herdc	true	en_US
pubs.publication-status	Published	en_US
pubs.volume	5	en_US

Abstract:

Type 1 diabetes (T1D) is caused by the autoimmune destruction of the insulin-producing pancreatic β-cells. People with T1D manage their hyperglycemia using daily insulin injections; however, this does not prevent the development of long-term diabetic complications such as retinopathy, nephropathy, neuropathy, and various macrovascular disorders. Currently, the only "cure" for T1D is pancreas transplantation or islet-cell transplantation; however, this is hampered by the limited number of donors and the requirement for life-long immunosuppression. As a result, the need for alternative therapies is vital. One of the strategies employed to correct T1D is the use of gene transfer to generate the production of an “artificial” β-cell that is capable of secreting insulin in response to fluctuating glucose concentrations that normally occurs in people without T1D. The treatment of many diseases using cell and gene therapy is generating significant attention in the T1D research community; however, for a cell therapy to enter clinical trials, success and safety must first be shown in an appropriate animal model. Animal models have been used in diabetes research for over a century, have improved our understanding of the pathophysiology of diabetes, and have led to the discovery of useful drugs for the treatment of the disease. Currently, the nonobese diabetic mouse is the animal model of choice for the study of T1D as it most closely reflects disease development in humans. The aim of this review is to evaluate the success of cell and gene therapy to reverse T1D in animal models for future clinical application.

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http://hdl.handle.net/10453/32759