Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies

2019 ◽  
pp. 17-28
Author(s):  
Hatice Burcu Şişli ◽  
Taha Bartu Hayal ◽  
Selin Seçkin ◽  
Selinay Şenkal ◽  
Binnur Kıratlı ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells ◽  
Recent Advances

scholarly journals Silent IL2RG Gene Editing in Human Pluripotent Stem Cells

2016 ◽  
Vol 24 (3) ◽  
pp. 582-591 ◽  
Author(s):  
Li B Li ◽  
Chao Ma ◽  
Geneve Awong ◽  
Marion Kennedy ◽  
German Gornalusse ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells ◽  
Il2rg Gene

How can gene-editing of human pluripotent stem cells help cystic fibrosis?

2019 ◽  
Author(s):  
Sara Cuevas Ocana ◽  
Amy Wong ◽  
Magomet Aushev ◽  
Jin Ye Yang ◽  
Neil Perkins ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells

scholarly journals Comprehensive Protocols for CRISPR/Cas9-based Gene Editing in Human Pluripotent Stem Cells

2016 ◽  
pp. 5B.6.1-5B.6.60 ◽  
Author(s):  
David P. Santos ◽  
Evangelos Kiskinis ◽  
Kevin Eggan ◽  
Florian T. Merkle
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells

scholarly journals Scarless Enriched selection of Genome edited Human Pluripotent Stem Cells Using Induced Drug Resistance

2019 ◽  
Author(s):  
Keun-Tae Kim ◽  
Ju-Chan Park ◽  
Haeseung Lee ◽  
Hyeon-Ki Jang ◽  
Yan Jin ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells ◽  
Cancer Drug ◽  
Efficient Gene ◽  
Anti Cancer ◽  
Low Efficiency ◽  
High Cytotoxicity ◽  
Selection Of

SummaryAn efficient gene editing technique for use in human pluripotent stem cells (hPSCs) would have great potential value in regenerative medicine, as well as in drug discovery based on isogenic human disease models. However, the extremely low efficiency of gene editing in hPSCs is a major technical hurdle that remains to be resolved. Previously, we demonstrated that YM155, a survivin inhibitor developed as an anti-cancer drug, induces highly selective cell death in undifferentiated hPSCs. In this study, we demonstrated that the high cytotoxicity of YM155 in hPSCs, which is mediated by selective cellular uptake of the drug, is due to high expression of SLC35F2 in these cells. Consistent with this, knockout of SLC35F2 with CRISPR-Cas9 or depletion with siRNAs made hPSCs highly resistant to YM155. Simultaneous gene editing of a gene of interest and transient knockdown of SLC35F2 following YM155 treatment enabled genome-edited hPSCs to survive because YM155 resistance was temporarily induced, thereby achieving enriched selection of genome-edited clonal populations. This precise and efficient genome editing approach took as little as 3 weeks without cell sorting or introduction of additional genes.

scholarly journals P74CCBE1 role along cardiac commitment of human pluripotent stem cells: exploring gene editing tools on loss-of-function studies

2018 ◽  
Vol 114 (suppl_1) ◽  
pp. S20-S20
Author(s):  
M Marques Silva ◽  
P Gomes-Alves ◽  
P Vicente ◽  
A Terrasso ◽  
J M Inacio ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells ◽  
Loss Of Function

scholarly journals The recent advances in the mathematical modelling of human pluripotent stem cells

2020 ◽  
Vol 2 (2) ◽  
Author(s):  
L. E. Wadkin ◽  
S. Orozco-Fuentes ◽  
I. Neganova ◽  
M. Lako ◽  
A. Shukurov ◽  
...  
Keyword(s):  
Stem Cells ◽  
Mathematical Modelling ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Recent Advances

Decoding the Epigenetic Heterogeneity of Human Pluripotent Stem Cells with Seamless Gene Editing

2016 ◽  
pp. 153-169
Author(s):  
Amar M. Singh ◽  
Dustin W. Perry ◽  
Valeriya V. Adjan Steffey ◽  
Kenneth Miller ◽  
Daniel W. Allison
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells
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