The complexity of dystrophin transcription and processing: implications of transcript imbalance on dystrophin gene targeting strategies.

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Title: The complexity of dystrophin transcription and processing: implications of transcript imbalance on dystrophin gene targeting strategies.
Authors: Rossi R; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK., Aartsma-Rus A; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands., Muntoni F; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK., Goyenvalle A; Université Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France. Electronic address: aurelie.goyenvalle@uvsq.fr., Spitali P; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands. Electronic address: p.spitali@lumc.nl.
Source: Neuromuscular disorders : NMD [Neuromuscul Disord] 2026 Feb; Vol. 59, pp. 106306. Date of Electronic Publication: 2025 Dec 13.
Publication Type: Journal Article; Review
Journal Info: Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Rossi+R%22">Rossi R</searchLink>; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Aartsma-Rus+A%22">Aartsma-Rus A</searchLink>; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Muntoni+F%22">Muntoni F</searchLink>; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Goyenvalle+A%22">Goyenvalle A</searchLink>; Université Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France. Electronic address: aurelie.goyenvalle@uvsq.fr.<br /><searchLink fieldCode="AU" term="%22Spitali+P%22">Spitali P</searchLink>; Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands. Electronic address: p.spitali@lumc.nl.
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  Data: <searchLink fieldCode="JN" term="%229111470%22">Neuromuscular disorders : NMD</searchLink> [Neuromuscul Disord] 2026 Feb; Vol. 59, pp. 106306. <i>Date of Electronic Publication: </i>2025 Dec 13.
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        Value: 10.1016/j.nmd.2025.106306
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        Text: English
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              Text: 2026 Feb
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