Progress in and Prospects of Genome Editing Tools for Human Disease Model Development and Therapeutic Applications

Hong Thi Lam Phan, Kyoungmi Kim, Ho Lee, Je Kyung Seong

Research output: Contribution to journalReview articlepeer-review

3 Citations (Scopus)


Programmable nucleases, such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas, are widely accepted because of their diversity and enormous potential for targeted genomic modifications in eukaryotes and other animals. Moreover, rapid advances in genome editing tools have accelerated the ability to produce various genetically modified animal models for studying human diseases. Given the advances in gene editing tools, these animal models are gradually evolving toward mimicking human diseases through the introduction of human pathogenic mutations in their genome rather than the conventional gene knockout. In the present review, we summarize the current progress in and discuss the prospects for developing mouse models of human diseases and their therapeutic applications based on advances in the study of programmable nucleases.

Original languageEnglish
Article number483
Issue number2
Publication statusPublished - 2023 Feb

Bibliographical note

Funding Information:
This work was supported by the Bio & Medical Technology Development Program of the National Research Foundation (NRF) funded by the Korean government (NRF-2020M3A9D5A01082439 and 2019R1A2C2087198 to K.K., 2014M3A9D5A01075128 to H.L. and 2013M3A9D5072550 and 2021M3H9A1030158 to J.K.S.).

Publisher Copyright:
© 2023 by the authors.


  • genome editing
  • mouse model of human disease
  • programmable nuclease
  • therapeutic application

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)


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