scholarly journals CRISPR/Cas9-based Targeted Genome Editing for the Development of Monogenic Diseases Models with Human Pluripotent Stem Cells

2018 ◽  
Vol 45 (1) ◽  
pp. e50 ◽  
Author(s):  
Navin Gupta ◽  
Koichiro Susa ◽  
Yoko Yoda ◽  
Joseph V. Bonventre ◽  
M. Todd Valerius ◽  
...  
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Monogenic Diseases

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.

scholarly journals Scarless Genome Editing of Human Pluripotent Stem Cells via Transient Puromycin Selection

2018 ◽  
Vol 10 (2) ◽  
pp. 642-654 ◽  
Author(s):  
Benjamin Steyer ◽  
Qian Bu ◽  
Evan Cory ◽  
Keer Jiang ◽  
Stella Duong ◽  
...  
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Puromycin Selection

scholarly journals Small molecules promote CRISPR-Cpf1-mediated genome editing in human pluripotent stem cells

2018 ◽  
Vol 9 (1) ◽  
Author(s):  
Xiaojie Ma ◽  
Xi Chen ◽  
Yan Jin ◽  
Wenyan Ge ◽  
Weiyun Wang ◽  
...  
Keyword(s):  
Stem Cells ◽  
Small Molecules ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells

scholarly journals A Cut above the Rest: Targeted Genome Editing Technologies in Human Pluripotent Stem Cells

2013 ◽  
Vol 289 (8) ◽  
pp. 4594-4599 ◽  
Author(s):  
Mo Li ◽  
Keiichiro Suzuki ◽  
Na Young Kim ◽  
Guang-Hui Liu ◽  
Juan Carlos Izpisua Belmonte
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells

scholarly journals Gene delivery methods and genome editing of human pluripotent stem cells

2019 ◽  
Vol 24 (2) ◽  
pp. 180-187 ◽  
Author(s):  
Patrycja Czerwińska ◽  
Sylwia Mazurek ◽  
Iga Kołodziejczak ◽  
Maciej Wiznerowicz
Keyword(s):  
Stem Cells ◽  
Gene Delivery ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Delivery Methods

Efficient scarless genome editing in human pluripotent stem cells

2018 ◽  
Vol 15 (12) ◽  
pp. 1045-1047 ◽  
Author(s):  
Kazuya Ikeda ◽  
Nobuko Uchida ◽  
Toshinobu Nishimura ◽  
Joseph White ◽  
Renata M. Martin ◽  
...  
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells
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