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A beginner's guide to gene editing.

Patrick T Harrison1, Stephen Hart2

  • 1Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.

Experimental Physiology
|December 29, 2017
PubMed
Summary
This summary is machine-generated.

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Gene editing technology has evolved significantly since the 1980s, with CRISPR-Cas9 now a widely accessible tool. Recent advances focus on improving efficiency and reducing off-target effects for clinical applications.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Genome editing allows precise modification of DNA in living cells.
  • Early gene editing techniques were limited in scope and efficiency.
  • The development of targeted nucleases revolutionized the field.

Purpose of the Study:

  • To summarize the historical development of gene editing technologies.
  • To highlight key advancements in gene editing, particularly CRISPR-Cas9.
  • To discuss improvements in efficiency and safety for gene editing.

Main Methods:

  • Review of early proof-of-concept studies.
  • Analysis of programmable nucleases, including zinc finger nucleases (ZFNs) and TAL effector nucleases (TALENs).
  • Focus on the development and impact of CRISPR-Cas9/guide RNA systems.
Keywords:
CRISPRCas9TAL-effector nucleasecystic fibrosisgene editingguide RNAzinc finger nuclease

Related Experiment Videos

Main Results:

  • Gene editing advanced from limited mouse models to broad applications in various cell types.
  • Zinc finger nucleases (ZFNs) and TAL effector nucleases (TALENs) improved specificity and ease of use.
  • CRISPR-Cas9/guide RNA, developed in 2013, made gene editing a readily accessible research tool.

Conclusions:

  • CRISPR-Cas9 has dramatically increased the accessibility and application of gene editing.
  • Ongoing research focuses on enhancing efficiency and minimizing off-target effects.
  • Gene editing is poised for clinical trials, signaling a new era in genetic medicine.