Journal
FRONTIERS IN ENDOCRINOLOGY
Volume 11, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fendo.2020.576632
Keywords
genome editing; beta cell; genome-wide association studies; maturity onset of diabetes of the young; stem cells; mouse models
Categories
Funding
- Wellcome Trust [WT098424AIA, 212625/Z/18/Z]
- MRC Programme [MR/R022259/1, MR/J0003042/1, MR/L020149/1]
- Experimental Challenge Grant (DIVA) [MR/L02036X/1]
- MRC [MR/N00275X/1]
- Diabetes UK [BDA/11/0004210, BDA/15/0005275, BDA 16/0005485]
- Imperial Confidence in Concept (ICiC)
- Royal Society Wolfson Research Merit Award
- European Union's Horizon 2020 research and innovation programme via the Innovative Medicines Initiative 2 Joint Undertaking [115881]
- European Union
- EFPIA
- BBSRC [BB/J015873/1] Funding Source: UKRI
- MRC [MR/R010676/1, MR/M012646/1, MR/L020149/1, MR/L02036X/1, MR/R022259/1, MR/N00275X/1, MR/K001981/1, MR/N020472/1] Funding Source: UKRI
- Medical Research Council [MR/N00275X/1] Funding Source: researchfish
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The inheritance of variants that lead to coding changes in, or the mis-expression of, genes critical to pancreatic beta cell function can lead to alterations in insulin secretion and increase the risk of both type 1 and type 2 diabetes. Recently developed clustered regularly interspaced short palindromic repeats (CRISPR/Cas9) gene editing tools provide a powerful means of understanding the impact of identified variants on cell function, growth, and survival and might ultimately provide a means, most likely after the transplantation of genetically corrected cells, of treating the disease. Here, we review some of the disease-associated genes and variants whose roles have been probed up to now. Next, we survey recent exciting developments in CRISPR/Cas9 technology and their possible exploitation for beta cell functional genomics. Finally, we will provide a perspective as to how CRISPR/Cas9 technology may find clinical application in patients with diabetes.
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