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World J Stem Cells. 2019 Nov 26;11(11):937-956. doi: 10.4252/wjsc.v11.i11.937.

CRISPR/Cas system: An emerging technology in stem cell research.

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Department of Medicine, Section of Internal Medicine D, University of Verona, Verona 37134, Italy.
Department of Biochemistry, University of Oxford, Oxford OX1 3QU, United Kingdom.
Department of Medicine, Section of Internal Medicine D, University of Verona, Verona 37134, Italy.
Department of Neurosciences, Biomedicine and Movement Sciences, Laboratory of Molecular Biology, Verona 37134, Italy.


The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix. The ability to modify selectively specific genes provides a powerful tool for characterizing gene functions, performing gene therapy, correcting specific genetic mutations, eradicating diseases, engineering cells and organisms to achieve new and different functions and obtaining transgenic animals as models for studying specific diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 technology has recently revolutionized genome engineering. The application of this new technology to stem cell research allows disease models to be developed to explore new therapeutic tools. The possibility of translating new systems of molecular knowledge to clinical research is particularly appealing for addressing degenerative diseases. In this review, we describe several applications of CRISPR/Cas9 to stem cells related to degenerative diseases. In addition, we address the challenges and future perspectives regarding the use of CRISPR/Cas9 as an important technology in the medical sciences.


CRISPR/Cas9; Degenerative diseases; Gene editing; Stem cells

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Conflict-of-interest statement: Authors declare no conflict of interests for this article.

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