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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 | 2020-01-01

SECONDARY ACCESSION(S): NCT03655678

REPOSITORIES: biostudies

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