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)

Abstract

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
JournalGenes
Volume14
Issue number2
DOIs
Publication statusPublished - 2023 Feb

Bibliographical note

Publisher Copyright:
© 2023 by the authors.

Keywords

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

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)

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