Genome Editing Overview

Principles

Definition

Genome editing is a set of molecular technologies developed for the targeted modification of the genetic material within living cells or organisms. This field encompasses various methods that enable precise alterations to DNA sequences, serving as a fundamental tool for biological research, therapeutic development, and biotechnological applications.

Ontological type

Core Methods

Molecular Mechanisms

Therapeutic Applications

Key developments

Key Figures

Recombinant and Cloning Infrastructure era

Thomas Tuschl [1] was active at Rockefeller University [3] and the Massachusetts Institute of Technology [4] during this era. His key contribution was the duplexes of 21-nucleotide RNAs mediating RNA interference in cultured mammalian cells [7], which established RNAi as a powerful tool for gene function interrogation and laid groundwork for genome-scale screening in this era. Thomas A. Kunkel [2] was at Stanford University [5] and The University of Texas Southwestern Medical Center [6] during this era. His key contribution was rapid and efficient site-specific mutagenesis without phenotypic selection [8], which advanced directed mutagenesis and genome engineering approaches central to the era's cloning infrastructure.

Programmable Nuclease Revolution era

Feng Zhang [1] is a leading figure at Harvard University [3] and the Institute of Cognitive and Brain Sciences [4] during the Programmable Nuclease Revolution era. His contributions include multiplex genome engineering using CRISPR/Cas systems [7] and in vivo genome editing using Staphylococcus aureus Cas9 [8], which advanced programmable nuclease platforms by enabling multiplexed targeting and in vivo edits. Luciano A. Marraffini [2] is a prominent figure at Rockefeller University [5] and Northwestern University [6] during this era. His key contribution in this era centers on co-authoring the 2013 Multiplex Genome Engineering Using CRISPR/Cas Systems [7], which demonstrated programmable targeting in diverse systems and seeded widespread adoption of genome edits for functional genomics.

Precision DSB‑Free Editing era

David R. Liu [1] is associated with Harvard University [3] and Université de Lille [4] during this era. His key contributions include the 2018 Predictable and precise template-free CRISPR editing of pathogenic variants [8], the 2019 Search-and-replace genome editing without double-strand breaks or donor DNA [6], and the 2020 Prime genome editing in rice and wheat [7], which collectively advanced the era by enabling precise, DSB-free editing and broad translational potential. Gregory A. Newby [2] is associated with Harvard University [3] and Massachusetts Institute of Technology [5] during this period. His sole listed contribution in the era is the 2019 paper 'Search-and-replace genome editing without double-strand breaks or donor DNA' [6], a pivotal demonstration of template-free editing that helped catalyze the precision DSB-free editing paradigm.