scholarly journals Introducing Point Mutations into Human Pluripotent Stem Cells using Seamless Genome Editing

10.3791/61152 ◽  
2020 ◽  
Author(s):  
Yu Wang ◽  
Andrew J. H. Smith ◽  
David C. Hay
Keyword(s):  
Stem Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Point Mutations ◽  
Human Pluripotent Stem Cells

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 The Impact of Acquired Genetic Abnormalities on the Clinical Translation of Human Pluripotent Stem Cells

Cells ◽  
2021 ◽  
Vol 10 (11) ◽  
pp. 3246
Author(s):  
Alexander Keller ◽  
Claudia Spits
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Chromosomal Abnormalities ◽  
Current Knowledge ◽  
Point Mutations ◽  
Human Pluripotent Stem Cells ◽  
Clinical Translation ◽  
Copy Number Changes ◽  
Clinical Potential ◽  
The Impact

Human pluripotent stem cells (hPSC) are known to acquire chromosomal abnormalities, which range from point mutations to large copy number changes, including full chromosome aneuploidy. These aberrations have a wide-ranging influence on the state of cells, in both the undifferentiated and differentiated state. Currently, very little is known on how these abnormalities will impact the clinical translation of hPSC, and particularly their potential to prime cells for oncogenic transformation. A further complication is that many of these abnormalities exist in a mosaic state in culture, which complicates their detection with conventional karyotyping methods. In this review we discuss current knowledge on how these aberrations influence the cell state and how this may impact the future of research and the cells’ clinical potential.

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 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 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
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