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Updated: Mar 25, 2026

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

Tandem repeat knockout utilizing the CRISPR/Cas9 system in human cells.

Qingyan Lv¹, Liangxue Lai¹, Lin Yuan¹

¹Jilin Provincial Key Laboratory of Animal Embryo Engineering, Jilin University, Changchun, 130062, China.

|February 14, 2016

Summary

This summary is machine-generated.

This study demonstrates generating large deletions in tandem repeats using CRISPR/Cas9 off-target effects. This method aids in studying the role of tandem repeats in genetic diseases and genome instability.

Keywords:

CRISPR/Cas9 Off-target mutation Regional deletions Tandem repeats

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Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

Published on: April 10, 2018

Area of Science:

Genetics
Molecular Biology
Genomics

Background:

Tandem repeats are significant contributors to human genetic diseases, genome variation, and instability.
Previous methods for regional deletions using CRISPR/Cas9 have limitations.

Purpose of the Study:

To explore generating regional deletions of tandem repeats by leveraging CRISPR/Cas9 off-target effects.
To investigate the feasibility of large-fragment deletions in tandem repeats within specific genes.

Main Methods:

Utilized a CRISPR/Cas9 system with single-guide RNAs (sgRNAs) in 293FT cells.
Exploited off-target effects of CRISPR/Cas9 to induce deletions.
Targeted tandem repeats within the MAGEL2 and XIST genes.

Main Results:

Successfully generated large-fragment deletions of tandem repeats.
Demonstrated the possibility of deleting tandem repeats located in the MAGEL2 and XIST genes.
Confirmed the efficacy of the sgRNA-directed CRISPR/Cas9 system for this purpose.

Conclusions:

Large-fragment deletions of tandem repeats can be achieved using a sgRNA-directed CRISPR/Cas9 system.
This approach facilitates future functional studies of tandem repeats.
The findings contribute to understanding genome variation and instability.