CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: A path to clinical success?

Gerace, D; Martiniello-Wilks, R; Nassif, NT; Lal, S; Steptoe, R; Simpson, AM

CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: A path to clinical success?

Gerace, D

Martiniello-Wilks, R

Nassif, NT

Lal, S

Steptoe, R Simpson, AM

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Publication Type:: Journal Article
Citation:: Stem Cell Research and Therapy, 2017, 8 (1)
Issue Date:: 2017-03-09

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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	Nassif, NT https://orcid.org/0000-0003-2675-8322	en_US
dc.contributor.author	Lal, S https://orcid.org/0000-0002-0911-0850	en_US
dc.contributor.author	Steptoe, R	en_US
dc.contributor.author	Simpson, AM https://orcid.org/0000-0002-2249-5097	en_US
dc.date.issued	2017-03-09	en_US
dc.identifier.citation	Stem Cell Research and Therapy, 2017, 8 (1)	en_US
dc.identifier.uri	http://hdl.handle.net/10453/86103
dc.description.abstract	© 2017 The Author(s). Due to their ease of isolation, differentiation capabilities, and immunomodulatory properties, the therapeutic potential of mesenchymal stem cells (MSCs) has been assessed in numerous pre-clinical and clinical settings. Currently, whole pancreas or islet transplantation is the only cure for people with type 1 diabetes (T1D) and, due to the autoimmune nature of the disease, MSCs have been utilised either natively or transdifferentiated into insulin-producing cells (IPCs) as an alternative treatment. However, the initial success in pre-clinical animal models has not translated into successful clinical outcomes. Thus, this review will summarise the current state of MSC-derived therapies for the treatment of T1D in both the pre-clinical and clinical setting, in particular their use as an immunomodulatory therapy and targets for the generation of IPCs via gene modification. In this review, we highlight the limitations of current clinical trials of MSCs for the treatment of T1D, and suggest the novel clustered regularly interspaced short palindromic repeat (CRISPR) gene-editing technology and improved clinical trial design as strategies to translate pre-clinical success to the clinical setting.	en_US
dc.relation	http://purl.org/au-research/grants/nhmrc/352909
dc.relation	http://purl.org/au-research/grants/nhmrc/513100
dc.relation	http://purl.org/au-research/grants/nhmrc/APP1086256
dc.relation.ispartof	Stem Cell Research and Therapy	en_US
dc.relation.isbasedon	10.1186/s13287-017-0511-8	en_US
dc.subject.mesh	Islets of Langerhans	en_US
dc.subject.mesh	Humans	en_US
dc.subject.mesh	Diabetes Mellitus, Type 1	en_US
dc.subject.mesh	Mesenchymal Stem Cell Transplantation	en_US
dc.subject.mesh	Cell Differentiation	en_US
dc.subject.mesh	Mesenchymal Stromal Cells	en_US
dc.subject.mesh	Genetic Therapy	en_US
dc.subject.mesh	CRISPR-Cas Systems	en_US
dc.subject.mesh	Gene Editing	en_US
dc.subject.mesh	Mesenchymal Stem Cells	en_US
dc.title	CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: A path to clinical success?	en_US
dc.type	Journal Article
utslib.citation.volume	1	en_US
utslib.citation.volume	8	en_US
utslib.for	1004 Medical Biotechnology	en_US
utslib.for	0601 Biochemistry and Cell Biology	en_US
utslib.for	1101 Medical Biochemistry and Metabolomics	en_US
utslib.for	06 Biological Sciences	en_US
utslib.for	10 Technology	en_US
utslib.for	11 Medical and Health 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
dcterms.accessRights	This work is distributed under a Creative Commons Attribution license (CC BY) http://creativecommons.org/licenses/by/4.0
pubs.issue	1	en_US
pubs.publication-status	Published	en_US
pubs.volume	8	en_US

Abstract:

© 2017 The Author(s). Due to their ease of isolation, differentiation capabilities, and immunomodulatory properties, the therapeutic potential of mesenchymal stem cells (MSCs) has been assessed in numerous pre-clinical and clinical settings. Currently, whole pancreas or islet transplantation is the only cure for people with type 1 diabetes (T1D) and, due to the autoimmune nature of the disease, MSCs have been utilised either natively or transdifferentiated into insulin-producing cells (IPCs) as an alternative treatment. However, the initial success in pre-clinical animal models has not translated into successful clinical outcomes. Thus, this review will summarise the current state of MSC-derived therapies for the treatment of T1D in both the pre-clinical and clinical setting, in particular their use as an immunomodulatory therapy and targets for the generation of IPCs via gene modification. In this review, we highlight the limitations of current clinical trials of MSCs for the treatment of T1D, and suggest the novel clustered regularly interspaced short palindromic repeat (CRISPR) gene-editing technology and improved clinical trial design as strategies to translate pre-clinical success to the clinical setting.

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http://hdl.handle.net/10453/86103