scholarly journals Stem Cells for Modeling Human Disease

10.5772/63231 ◽  
2016 ◽  
Author(s):  
F. Martín ◽  
A. Sánchez-Gilabert ◽  
M. Tristán-Manzano ◽  
K. Benabdellah
Keyword(s):  
Stem Cells ◽  
Human Disease

scholarly journals Controlling homology-directed repair outcomes in human stem cells with dCas9

2021 ◽  
Author(s):  
William C Skarnes ◽  
Gang Ning ◽  
Sofia Giansiracusa ◽  
Alexander S Cruz ◽  
Cornelis Blauwendraat ◽  
...  
Keyword(s):  
Stem Cells ◽  
High Activity ◽  
Human Disease ◽  
Human Stem Cells ◽  
Single Nucleotide Variants ◽  
Single Nucleotide ◽  
Homology Directed Repair ◽  
Guide Rnas

Modeling human disease in human stem cells requires precise, scarless editing of single nucleotide variants (SNV) on one or both chromosomes. Here we describe improved conditions for Cas9 RNP editing of SNVs that yield high rates of biallelic homology-directed repair. To recover both heterozygous and homozygous SNV clones, catalytically inactive dCas9 was added to moderate high activity Cas9 RNPs. dCas9 can also block re-cutting and damage to SNV alleles engineered with non-overlapping guide RNAs.

scholarly journals Modeling human neurodevelopmental diseases with brain organoids

2022 ◽  
Vol 11 (1) ◽  
Author(s):  
Xiaoxiang Lu ◽  
Jiajie Yang ◽  
Yangfei Xiang
Keyword(s):  
Stem Cells ◽  
Human Brain ◽  
Pluripotent Stem Cells ◽  
Neurodevelopmental Disorders ◽  
Human Disease ◽  
Gene Editing ◽  
Disease Modeling ◽  
Developmental Abnormalities ◽  
The Past ◽  
Underlying Mechanisms

AbstractStudying the etiology of human neurodevelopmental diseases has long been a challenging task due to the brain’s complexity and its limited accessibility. Human pluripotent stem cells (hPSCs)-derived brain organoids are capable of recapitulating various features and functionalities of the human brain, allowing the investigation of intricate pathogenesis of developmental abnormalities. Over the past years, brain organoids have facilitated identifying disease-associated phenotypes and underlying mechanisms for human neurodevelopmental diseases. Integrating with more cutting-edge technologies, particularly gene editing, brain organoids further empower human disease modeling. Here, we review the latest progress in modeling human neurodevelopmental disorders with brain organoids.

scholarly journals Genetic Engineering of Mesenchymal Stem Cells and Its Application in Human Disease Therapy

2010 ◽  
Vol 21 (11) ◽  
pp. 1513-1526 ◽  
Author(s):  
Conrad P. Hodgkinson ◽  
José A. Gomez ◽  
Maria Mirotsou ◽  
Victor J. Dzau
Keyword(s):  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Genetic Engineering ◽  
Human Disease ◽  
Disease Therapy

scholarly journals Modelling Human Disease with Pluripotent Stem Cells

2013 ◽  
Vol 13 (2) ◽  
pp. 99-110 ◽  
Author(s):  
Richard Siller ◽  
Sebastian Greenhough ◽  
In-Hyun Park ◽  
Gareth J. Sullivan
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Human Disease
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