4.5 Review

Epigenome editing by CRISPR/Cas9 in clinical settings: possibilities and challenges

Journal

BRIEFINGS IN FUNCTIONAL GENOMICS
Volume 19, Issue 3, Pages 215-228

Publisher

OXFORD UNIV PRESS
DOI: 10.1093/bfgp/elz035

Keywords

epigenome editing; CRISPR/Cas9; clinical setting; diseases; challenge

Funding

  1. National Key R&D Program of China [2016YFC1000601]
  2. National Natural Science Funds [81571400, 81771662, 81771580, 81971381]

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Epigenome editing is a promising approach for both basic research and clinical application. With the convergence of techniques from different fields, regulating gene expression artificially becomes possible. From a clinical point of view, targeted epigenome editing by CRISPR/Cas9 of disease-related genes offers novel therapeutic avenues for many diseases. In this review, we summarize the EpiEffectors used in epigenome editing by CRISPR/Cas9, current applications of epigenome editing and progress made in this field. Moreover, application challenges such as off-target effects, inefficient delivery, stability and immunogenicity are discussed. In conclusion, epigenome editing by CRISPR/Cas9 has broad prospects in the clinic, and future work will promote the application of this technology.

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