Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing

doi:10.7150/thno.43360

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Theranostics 2020; 10(10):4374-4382. doi:10.7150/thno.43360 This issue Cite

Review

Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

Shao-Shuai Wu^1*, Qing-Cui Li^1*, Chang-Qing Yin¹, Wen Xue^2,3✉, Chun-Qing Song^1✉

1. Key Laboratory of Growth Regulation and Transformation Research of Zhejiang Province, School of Life Sciences, Westlake University, 18 Shilongshan Road, Hangzhou 310024, Zhejiang Province, China.
2. RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts
3. Program in Molecular Medicine and Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, Massachusetts
^*Equal contributors

Citation:

Wu SS, Li QC, Yin CQ, Xue W, Song CQ. Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. Theranostics 2020; 10(10):4374-4382. doi:10.7150/thno.43360. https://www.thno.org/v10p4374.htm

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Abstract

CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non-homologous end joining, homology- directed repair, and single-base exchanges. Cas9/Cas12a nuclease, dCas9 transcriptional regulators, base editors, PRIME editors and RNA editing tools are widely used in basic research. Currently, a variety of CRISPR/Cas-based therapeutics are being investigated in clinical trials. Among many new findings that have advanced the field, we highlight a few recent advances that are relevant to CRISPR/Cas-based gene therapies for monogenic human genetic diseases.

Keywords: CRISPR/Cas, Gene editing, Gene therapy, Human disease, Genetic disease

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APA

Wu, S.S., Li, Q.C., Yin, C.Q., Xue, W., Song, C.Q. (2020). Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. Theranostics, 10(10), 4374-4382. https://doi.org/10.7150/thno.43360.

ACS

Wu, S.S.; Li, Q.C.; Yin, C.Q.; Xue, W.; Song, C.Q. Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. Theranostics 2020, 10 (10), 4374-4382. DOI: 10.7150/thno.43360.

NLM

CSE

Wu SS, Li QC, Yin CQ, Xue W, Song CQ. 2020. Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases. Theranostics. 10(10):4374-4382.

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