REVIEW article
Front. Bioeng. Biotechnol.
Sec. Cell and Gene Therapy
Volume 13 - 2025 | doi: 10.3389/fbioe.2025.1562412
This article is part of the Research TopicCRISPR tailored gene editing – the real promise to treat human diseaseView all 7 articles
Harnessing CRISPR potential for Intervertebral Disc Regeneration Strategies
Provisionally accepted
- 1ICBAS, School of Medicine and Biomedical Sciences, Institute of Biomedical Sciences Abel Salazar, University of Porto, Porto, Portugal
- 2Instituto de Investigação e Inovação em Saúde, Universidade do Porto, Porto, Porto, Portugal
- 3Genethon, Évry, France
- 4INSERM U951 Approches Génétiques Intégrées et Découvertes Thérapeutiques pour les Maladies Rares, Evry, France
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Genome editing technologies, particularly CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), have broadened the possibilities of genetic research and molecular biology by enabling precise modifications of the genome, offering novel therapeutic potential for various disorders. Herein, we present an overview of traditional genome editing techniques and delve deeper into the CRISPR toolbox, with particular attention given to epigenetic and transcriptional regulation. In the context of the intervertebral disc (IVD), CRISPR offers an unprecedented approach to address the mechanisms underlying tissue degeneration, advancing the development of revolutionary therapies for Low Back Pain (LBP). As so, we showcase how to leverage CRISPR systems for IVD. This cuttingedge technology has been successfully used to improve our understanding of IVD biology through functional studies and disease modeling. Most relevant research prioritizes new targets associated with the extracellular matrix (ECM), pain sensing or inflammatory pathways. Promising CRISPR applications encompass IVD regeneration by recapitulation of a regenerative environment or by targeting important degenerative catalysts. In the future, priority should be given to fetal gene reactivation, multiple healthy gene expression enhancement and disease -associated polymorphisms' correction. Despite several challenges such as effective delivery, off-target effects, as well as ethical and safety concerns, exciting clinical trials are anticipated in the years to come, providing more effective and long-lasting solutions for IVD degeneration.
Keywords: CRISPR/Cas9, Intervertebral Disc, Low Back Pain, Degenerative disc disease, genome editing tools
Received: 17 Jan 2025; Accepted: 15 Apr 2025.
Copyright: © 2025 Milheiro, Moura, Amendola, Barbosa and Caldeira. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Joana Caldeira, Instituto de Investigação e Inovação em Saúde, Universidade do Porto, Porto, 4200-135, Porto, Portugal
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.