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.


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