IMR Press / FBL / Volume 27 / Issue 8 / DOI: 10.31083/j.fbl2708241
Open Access Review
Genome Editing in Translational Medicine: An Inventory
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1 Genome Engineering and Model Development lab (GEMD), IUF-Leibniz Research Institute for Environmental Medicine, 40225 Düsseldorf, Germany
*Correspondence: (Andrea Rossi)
These authors contributed equally.
Academic Editor: Said El Shamieh
Front. Biosci. (Landmark Ed) 2022, 27(8), 241;
Submitted: 24 June 2022 | Revised: 8 July 2022 | Accepted: 18 July 2022 | Published: 12 August 2022
Copyright: © 2022 The Author(s). Published by IMR Press.
This is an open access article under the CC BY 4.0 license.

Genomic mutations are the driving force of biological diversity but they are also the cause of a plethora of human diseases ranging from heritable disorders to neurological pathologies and cancer. For most genetic disorders, there is no curative treatment available to date. The demand for precise, preferably patient-specific, treatment regimen offering cure is naturally high. Genome editing by Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas enables targeted manipulation of genomes, thereby offering the opportunity to treat such diseases. While ethical and regulatory guidelines need to be developed and considered, the prospect of genome editing for curative treatment is certainly exciting. Here, we review the current state of therapeutics based on genome editing techniques. We highlight recent breakthroughs, describe clinical trials employing genome editing-based medicine, discuss the benefits and pitfalls, and take a look into the future of genome editing.

genome editing
prime editing
clinical application
Fig. 1.
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