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

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.

SUBMITTER: Wu SS

PROVIDER: S-EPMC7150498 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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

Wu Shao-Shuai SS Li Qing-Cui QC Yin Chang-Qing CQ Xue Wen W Song Chun-Qing CQ

Theranostics 20200315 10

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 ne ...[more]

PMID: 32292501

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