{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T19:51:53Z","timestamp":1791575513101,"version":"4.3.4"},"reference-count":148,"publisher":"MDPI AG","issue":"6","license":[{"start":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T00:00:00Z","timestamp":1779926400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Friends of Garrett Cumming Research Fund"},{"DOI":"10.13039\/501100000223","name":"Muscular Dystrophy Canada","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100000223","id-type":"DOI","asserted-by":"crossref"}]},{"id":[{"id":"https:\/\/ror.org\/000hkd473","id-type":"ROR","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000024","name":"Canadian Institute of Health Research","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Women and Children\u2019s Health Research Institute"},{"id":[{"id":"https:\/\/ror.org\/03e04kk51","id-type":"ROR","asserted-by":"publisher"}]},{"name":"HM Toupin Neurological Science Research Fund"},{"id":[{"id":"https:\/\/ror.org\/05ghs6f64","id-type":"ROR","asserted-by":"publisher"}]},{"name":"U.S. Department of Defense"},{"id":[{"id":"https:\/\/ror.org\/0447fe631","id-type":"ROR","asserted-by":"publisher"}]},{"name":"Heart and Stroke Foundation Canada"},{"id":[{"id":"https:\/\/ror.org\/00qbpyp73","id-type":"ROR","asserted-by":"publisher"}]},{"name":"Japanese Society of Neurology"},{"DOI":"10.13039\/100004312","name":"Eli Lilly and Company","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100004312","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Biomolecules"],"abstract":"<jats:p>RNA-based therapeutics are reshaping the treatment landscape for skeletal muscle disorders by enabling modulation of RNA processing or direct correction of disease-causing alleles. In Duchenne muscular dystrophy (DMD), four antisense oligonucleotides\u2014eteplirsen, golodirsen, viltolarsen, and casimersen\u2014have received FDA approval; these phosphorodiamidate morpholino oligomers (PMOs) induce exon skipping to restore the reading frame and enable expression of internally truncated dystrophin. Beyond splice switching, RNA therapeutics include RNase H-active gapmers and steric-blocking antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs) that mediate post-transcriptional gene silencing, and RNA-guided gene-modifying technologies such as CRISPR systems that can reframe or repair endogenous alleles. Despite major progress in DMD, broader clinical impact remains constrained by inefficient delivery to skeletal and especially cardiac muscle, the need for repeat administration for most modalities, and safety considerations that limit dose escalation and durability. Next-generation approaches aim to overcome these barriers through peptide- or antibody-conjugated oligonucleotides that enhance cellular uptake and tissue distribution, alternative chemistries with improved stability and potency, and viral or non-viral platforms for durable splice modulation. In parallel, CRISPR-based strategies\u2014including base and prime editing\u2014offer the prospect of one-time correction, while raising important questions regarding delivery, immunogenicity, editing specificity, and long-term safety. This review synthesizes recent advances in antisense and gene-modifying strategies for skeletal muscle and highlights practical priorities for translation, including improved muscle\/heart delivery, controllable safety mechanisms, scalable manufacturing, and standardized biomarker-to-clinical outcome relationships.<\/jats:p>","DOI":"10.3390\/biom16060794","type":"journal-article","created":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T09:57:43Z","timestamp":1779962263000},"page":"794","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies"],"prefix":"10.3390","volume":"16","author":[{"given":"Takayuki","family":"Kuroda","sequence":"first","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6672-6742","authenticated-orcid":false,"given":"Toshifumi","family":"Yokota","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada"},{"name":"The Friends of Garrett Cumming Research & Muscular Dystrophy Canada, HM Toupin Neurological Sciences Research, Edmonton, AB T6G 2H7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,5,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"e114760","DOI":"10.15252\/embj.2023114760","article-title":"RNA-based Medicine: From Molecular Mechanisms to Therapy","volume":"42","author":"Sparmann","year":"2023","journal-title":"EMBO J."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"3802","DOI":"10.1016\/j.apsb.2024.05.008","article-title":"Current Status and Trends in Small Nucleic Acid Drug Development: Leading the Future","volume":"14","author":"Miao","year":"2024","journal-title":"Acta Pharm. 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