{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T01:26:30Z","timestamp":1788830790310,"version":"build-2803163510"},"reference-count":277,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2026,5,7]],"date-time":"2026-05-07T00:00:00Z","timestamp":1778112000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Defeat Duchenne Foundation","award":["53320215"],"award-info":[{"award-number":["53320215"]}]},{"name":"Vanier Canada Graduate Scholarship","award":["492510"],"award-info":[{"award-number":["492510"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cells"],"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) remains a major challenge in genetic medicine due to the difficulty of achieving durable, body-wide restoration of dystrophin in post-mitotic muscle tissues. Although current therapies\u2014including exon skipping and micro-dystrophin gene replacement\u2014have demonstrated clinical feasibility, their benefits are limited by incomplete efficacy, mutation specificity, and the need for repeated or high-dose interventions. These limitations highlight the need for strategies capable of directly and permanently correcting the underlying genetic defect. Recent advances in genome editing have positioned CRISPR-based technologies as promising candidates for this objective. Rather than functioning as a single approach, gene-editing platforms encompass a spectrum of strategies\u2014including exon deletion, exon reframing, base editing, and prime editing\u2014each with distinct advantages depending on the mutational context. In particular, the emergence of precision editing tools has enabled controlled nucleotide-level modifications, expanding the range of correctable mutations while reducing reliance on double-strand DNA breaks. In this review, we adopt a comparative and translational perspective to evaluate gene-editing strategies for DMD. We examine how different approaches align with specific mutation types, summarize key findings from preclinical studies, and analyze the major barriers to clinical implementation, including delivery efficiency, immune responses, editing durability, and genomic safety. We further discuss emerging innovations in editing technologies and delivery systems that aim to address these limitations. Collectively, this work reframes gene editing as a decision-oriented and application-driven therapeutic framework. Continued integration of advances in genome engineering, delivery platforms, and muscle biology will be essential to translate these technologies into safe, effective, and durable treatments capable of altering the clinical trajectory of DMD.<\/jats:p>","DOI":"10.3390\/cells15100852","type":"journal-article","created":{"date-parts":[[2026,5,7]],"date-time":"2026-05-07T14:05:46Z","timestamp":1778162746000},"page":"852","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9691-1364","authenticated-orcid":false,"given":"Ayesha","family":"Siddika","sequence":"first","affiliation":[{"name":"D\u00e9partement de M\u00e9decine Mol\u00e9culaire, Universit\u00e9 Laval, Qu\u00e9bec, QC G1V 0A6, Canada"},{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jo\u00ebl","family":"Rousseau","sequence":"additional","affiliation":[{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"F\u00e9lix","family":"Veillette","sequence":"additional","affiliation":[{"name":"D\u00e9partement de M\u00e9decine Mol\u00e9culaire, Universit\u00e9 Laval, Qu\u00e9bec, QC G1V 0A6, Canada"},{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8264-4090","authenticated-orcid":false,"given":"Camille","family":"Bouchard","sequence":"additional","affiliation":[{"name":"D\u00e9partement de M\u00e9decine Mol\u00e9culaire, Universit\u00e9 Laval, Qu\u00e9bec, QC G1V 0A6, Canada"},{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8239-2279","authenticated-orcid":false,"given":"Yaoyao","family":"Lu","sequence":"additional","affiliation":[{"name":"D\u00e9partement de M\u00e9decine Mol\u00e9culaire, Universit\u00e9 Laval, Qu\u00e9bec, QC G1V 0A6, Canada"},{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9404-9195","authenticated-orcid":false,"given":"Jacques P.","family":"Tremblay","sequence":"additional","affiliation":[{"name":"D\u00e9partement de M\u00e9decine Mol\u00e9culaire, Universit\u00e9 Laval, Qu\u00e9bec, QC G1V 0A6, Canada"},{"name":"Centre de Recherche du Centre Hospitalier Universitaire de Qu\u00e9bec, Qu\u00e9bec, QC G1E 6W2, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,5,7]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"7","author":"Duan","year":"2021","journal-title":"Nat. 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