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

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

doi:10.7150/thno.43360

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

Theranostics. 2020 Mar 15;10(10):4374-4382. doi: 10.7150/thno.43360. eCollection 2020.

Authors

Shao-Shuai Wu¹, Qing-Cui Li¹, Chang-Qing Yin¹, Wen Xue^{2

3}, Chun-Qing Song¹

Affiliations

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.

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; Genetic disease; Human disease.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Animals
CRISPR-Cas Systems*
Cell Line
Gene Editing*
Genetic Diseases, Inborn / therapy*
Genetic Therapy*
Humans

Abstract

Publication types

MeSH terms

Grants and funding