CRISPR/Cas9 Genome Editing: A Promising Tool for Therapeutic Applications of Induced Pluripotent Stem Cells

Author(s): Yanli Zhang, Danuta Sastre, Feng Wang*.

Journal Name: Current Stem Cell Research & Therapy

Volume 13 , Issue 4 , 2018

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Background: Induced pluripotent stem cells hold tremendous potential for biological and therapeutic applications. The development of efficient technologies for targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9) system is recently recognized as a powerful tool for editing DNA at specific loci.

Objective: The ease of use of the CRISPR-Cas9 technology will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. More recently, this system was modified to repress (CRISPR interference, CRISPRi) or activate (CRISPR activation, CRISPRa) gene expression without alterations in the DNA, which amplified the scope of applications of CRISPR systems for stem cell biology.

Results and Conclusion: Here, we highlight latest advances of CRISPR-associated applications in human pluripotent stem cells. The challenges and future prospects of CRISPR-based systems for human research are also discussed.

Keywords: CRISPR/Cas9, CRISPRi, CRISPRa, pluripotent stem cells, iPSC, therapy.

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Article Details

Year: 2018
Page: [243 - 251]
Pages: 9
DOI: 10.2174/1574888X13666180214124800
Price: $65

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