Research map: Duchenne muscular dystrophy: from gene to gene-ius therapies

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  1. Dystrophin: The protein product of the duchenne muscular dystrophy locus · Eric P. Hoffman · 1987 · 4743 citations · Cited by this paper
  2. Repairing the defective folding of alpha-sarcoglycan is needed to promote myogenic cell engraftment in 3D artificial muscle models of LGMDR3 · 2025 · Related
  3. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management · Katharine M. D. Bushby · 2009 · 2080 citations · Cited by this paper
  4. Abnormalities in the genioglossus muscle and its neuromuscular synapse in leptin-deficient male mice · 2025 · Related
  5. X chromosome-linked muscular dystrophy (mdx) in the mouse. · Grahame Bulfield · 1984 · 1789 citations · Cited by this paper
  6. Fighting for every beat: cardiac therapies in Duchenne muscular dystrophy · 2025 · Related
  7. The muscular dystrophies · Alan E H Emery · 2002 · 1677 citations · Cited by this paper
  8. Ephrin-A5 or EphA7 stimulation is anti-proliferative for human rhabdomyosarcoma in vitro · 2025 · Related
  9. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. · Basil J. Petrof · 1993 · 1550 citations · Cited by this paper
  10. The metabolic role of corticotropin-releasing hormone receptor 2 and its UCN peptides: emerging therapeutic potential · 2025 · Related
  11. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein · M. Koenig · 1988 · 1545 citations · Cited by this paper
  12. Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans · 2026 · Related
  13. Asymmetric Self-Renewal and Commitment of Satellite Stem Cells in Muscle · Shihuan Kuang · 2007 · 1457 citations · Cited by this paper
  14. The rise of rat models for Duchenne muscular dystrophy and therapeutic evaluations · 2025 · Related
  15. A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin · JM Ervasti · 1993 · 1405 citations · Cited by this paper
  16. Unleashing the Potential of Givinostat: A Novel Therapy for Duchenne Muscular Dystrophy · 2025 · Related
  17. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management · David J. Birnkrant · 2018 · 1391 citations · Cited by this paper
  18. Senescence dynamics define therapeutic windows for Duchenne muscular dystrophy in DBA/2-mdx mice. · 2025 · Related
  19. The role of lipid components in lipid nanoparticles for vaccines and gene therapy · Camilla Hald Albertsen · 2022 · 1189 citations · Cited by this paper
  20. A 3D skeletal muscle system for disease modelling and secretome profiling of Duchenne muscular dystrophy. · 2025 · Related
  21. Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle · James M. Ervasti · 1990 · 1037 citations · Cited by this paper
  22. Utrophin-Dystrophin-Deficient Mice as a Model for Duchenne Muscular Dystrophy · Anne E. Deconinck · 1997 · 752 citations · Cited by this paper
  23. MyoD is required for myogenic stem cell function in adult skeletal muscle. · Lynn A. Megeney · 1996 · 724 citations · Cited by this paper
  24. Skeletal and Cardiac Myopathies in Mice Lacking Utrophin and Dystrophin: A Model for Duchenne Muscular Dystrophy · Ronald Mark Grady · 1997 · 646 citations · Cited by this paper
  25. Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division · Nicolas A. Dumont · 2015 · 631 citations · Cited by this paper
  26. Systemic delivery of genes to striated muscles using adeno-associated viral vectors · Paul Gregorevic · 2004 · 623 citations · Cited by this paper
  27. Expression of full-length utrophin prevents muscular dystrophy in mdx mice · Jonathon M. Tinsley · 1998 · 616 citations · Cited by this paper
  28. Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy · Scott Q. Harper · 2002 · 582 citations · Cited by this paper
  29. Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species · Mohammadsharif Tabebordbar · 2021 · 539 citations · Cited by this paper
  30. Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy · Jerry R. Mendell · 1995 · 532 citations · Cited by this paper
  31. Short Telomeres and Stem Cell Exhaustion Model Duchenne Muscular Dystrophy in mdx/mTR Mice · Alessandra Sacco · 2010 · 523 citations · Cited by this paper
  32. Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy · Dongsheng Duan · 2018 · 506 citations · Cited by this paper
  33. Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice · Radbod Darabi · 2012 · 482 citations · Cited by this paper
  34. Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy · David G. Ousterout · 2015 · 474 citations · Cited by this paper
  35. In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation · Hsin‐Kai Liao · 2017 · 463 citations · Cited by this paper
  36. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy · Christopher E. Nelson · 2019 · 446 citations · Cited by this paper
  37. Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca 2+ , Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy · David G. Allen · 2015 · 445 citations · Cited by this paper
  38. mdx mice show progressive weakness and muscle deterioration with age · Christian Pastoret · 1995 · 413 citations · Cited by this paper
  39. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells · Courtney S. Young · 2016 · 395 citations · Cited by this paper

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