scholarly journals Highly Efficient CRISPR-Cas9-Mediated Genome Editing in Human Pluripotent Stem Cells

2018 ◽  
Vol 48 (1) ◽  
pp. e64 ◽  
Author(s):  
Jean Ann Maguire ◽  
Fabian L. Cardenas-Diaz ◽  
Paul Gadue ◽  
Deborah L. French
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Highly Efficient

scholarly journals Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

2019 ◽  
Vol 24 (5) ◽  
pp. 821-828.e5 ◽  
Author(s):  
Renata M. Martin ◽  
Kazuya Ikeda ◽  
M. Kyle Cromer ◽  
Nobuko Uchida ◽  
Toshinobu Nishimura ◽  
...  
Keyword(s):  
Stem Cells ◽  
Homologous Recombination ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Highly Efficient ◽  
Marker Free

scholarly journals Highly efficient genome editing via CRISPR–Cas9 in human pluripotent stem cells is achieved by transient BCL-XL overexpression

2018 ◽  
Vol 46 (19) ◽  
pp. 10195-10215 ◽  
Author(s):  
Xiao-Lan Li ◽  
Guo-Hua Li ◽  
Juan Fu ◽  
Ya-Wen Fu ◽  
Lu Zhang ◽  
...  
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Highly Efficient

scholarly journals An iCRISPR Platform for Rapid, Multiplexable, and Inducible Genome Editing in Human Pluripotent Stem Cells

2014 ◽  
Vol 15 (2) ◽  
pp. 215-226 ◽  
Author(s):  
Federico González ◽  
Zengrong Zhu ◽  
Zhong-Dong Shi ◽  
Katherine Lelli ◽  
Nipun Verma ◽  
...  
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells

scholarly journals Genome Editing Human Pluripotent Stem Cells to Model β-Cell Disease and Unmask Novel Genetic Modifiers

2021 ◽  
Vol 12 ◽  
Author(s):  
Matthew N. George ◽  
Karla F. Leavens ◽  
Paul Gadue
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Genetic Disease ◽  
Single Gene ◽  
Monogenic Diabetes ◽  
Human Pluripotent Stem Cells ◽  
Genetic Modifiers ◽  
Β Cell ◽  
Protein Coding

A mechanistic understanding of the genetic basis of complex diseases such as diabetes mellitus remain elusive due in large part to the activity of genetic disease modifiers that impact the penetrance and/or presentation of disease phenotypes. In the face of such complexity, rare forms of diabetes that result from single-gene mutations (monogenic diabetes) can be used to model the contribution of individual genetic factors to pancreatic β-cell dysfunction and the breakdown of glucose homeostasis. Here we review the contribution of protein coding and non-protein coding genetic disease modifiers to the pathogenesis of diabetes subtypes, as well as how recent technological advances in the generation, differentiation, and genome editing of human pluripotent stem cells (hPSC) enable the development of cell-based disease models. Finally, we describe a disease modifier discovery platform that utilizes these technologies to identify novel genetic modifiers using induced pluripotent stem cells (iPSC) derived from patients with monogenic diabetes caused by heterozygous mutations.

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