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


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. © The author(s).

Authors Wu SS, Li QC, Yin CQ, Xue W, Song CQ
Journal Theranostics
Publication Date 2020;10(10):4374-4382
PubMed 32292501
PubMed Central PMC7150498
DOI 10.7150/thno.43360

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