Research map: Safety, Tolerability, and Efficacy of a Prophylactic Sirolimus Protocol for Patients Receiving Delandistrogene Moxeparvovec-Rokl Gene Therapy

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  1. 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
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  18. mTOR inhibitors lower an intrinsic barrier to virus infection mediated by IFITM3 · Guoli Shi · 2018 · 90 citations · Cited by this paper
  19. Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing · 2026 · Related
  20. Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial · Jerry R. Mendell · 2023 · 85 citations · Cited by this paper
  21. Gene Therapy in Patients with the Crigler–Najjar Syndrome · Lorenzo D’Antiga · 2023 · 83 citations · Cited by this paper
  22. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders · Besarte Vrellaku · 2024 · 71 citations · Cited by this paper
  23. Intrathecal Gene Therapy for Giant Axonal Neuropathy · Diana Xerxes Bharucha-Goebel · 2024 · 65 citations · Cited by this paper
  24. An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy · Laurence O. Whiteley · 2023 · 58 citations · Cited by this paper
  25. The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer · Zhiquan Xiang · 2022 · 32 citations · Cited by this paper
  26. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy · Craig Mitchell Zaidman · 2024 · 24 citations · Cited by this paper
  27. Systemic Toxicity of Recombinant Adeno-Associated Virus Gene Therapy Vectors · Basel T. Assaf · 2024 · 24 citations · Cited by this paper
  28. Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques · Alexander Kistner · 2023 · 12 citations · Cited by this paper
  29. Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis · Niccolo Maurizi · 2026 · 12 citations · Cited by this paper
  30. Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance · Beth D. Kaufman · 2024 · 10 citations · Cited by this paper
  31. Acute Liver Injury Following Delandistrogene Moxeparvovec Gene Therapy Requiring Intravenous Immunoglobulin · Ruthwik Duvuru · 2024 · 9 citations · Cited by this paper
  32. Reversal of Acute Liver Injury Following Delandistrogene Moxeparvovec Gene Therapy Using Sirolimus and Immunoglobulin · Serena M. Neumann · 2025 · 7 citations · Cited by this paper
  33. Hepatic Manifestations Following Gene Therapy · A. D. Roy · 2025 · 5 citations · Cited by this paper
  34. Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus · Maryam Oskoui · 2025 · 4 citations · Cited by this paper
  35. Safety of in vivo gene therapy in children: mechanisms and management of liver injury · Rebecca Jeyaraj · 2026 · 4 citations · Cited by this paper
  36. Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management · Perry B. Shieh · 2026 · 3 citations · Cited by this paper

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