Research map: Rewriting the sarcomere: Gene therapy approaches for hypertrophic cardiomyopathy from bench to bedside

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  1. Adeno-associated virus vector as a platform for gene therapy delivery · Dan Wang · 2019 · 2488 citations · Cited by this paper
  2. A single-cell solution to a transplant mystery · 2026 · Related
  3. A Mouse Model of Familial Hypertrophic Cardiomyopathy · Anja A. T. Geisterfer-Lowrance · 1996 · 560 citations · Cited by this paper
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  5. 2024 AHA/ACC/AMSSM/HRS/PACES/SCMR Guideline for the Management of Hypertrophic Cardiomyopathy · Steve R. Ommen · 2024 · 372 citations · Cited by this paper
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  7. Allele-Specific Silencing of Mutant Myh6 Transcripts in Mice Suppresses Hypertrophic Cardiomyopathy · Jianming Jiang · 2013 · 236 citations · Cited by this paper
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  11. A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8 · Virginie Monteilhet · 2011 · 206 citations · Cited by this paper
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  13. Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice · Daniel Reichart · 2023 · 199 citations · Cited by this paper
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  15. Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice · Andreas C. Chai · 2023 · 181 citations · Cited by this paper
  16. Building on CD19 based therapy for relapse or refractory follicular lymphoma · 2026 · Related
  17. Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions · Thomas P. Weber · 2021 · 161 citations · Cited by this paper
  18. Ethics of gene therapy · 2026 · Related
  19. Dystrophin Immunity after Gene Therapy for Duchenne’s Muscular Dystrophy · Carsten G. Bönnemann · 2023 · 127 citations · Cited by this paper
  20. Impact of lead-in ADT on grade ≥3 neutropenia in patients with metastatic hormone-sensitive prostate cancer treated with triplet therapy · 2026 · Related
  21. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy · Martin Schulz · 2023 · 126 citations · Cited by this paper
  22. Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice · Christina Gedicke‐Hornung · 2013 · 103 citations · Cited by this paper
  23. Capsid-specific removal of circulating antibodies to adeno-associated virus vectors · Bérangère Bertin · 2020 · 99 citations · Cited by this paper
  24. Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease · Barry Greenberg · 2024 · 91 citations · Cited by this paper
  25. Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics · William H. Bradford · 2023 · 76 citations · Cited by this paper
  26. Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption · Alejandro Orlowski · 2020 · 74 citations · Cited by this paper
  27. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders · Besarte Vrellaku · 2024 · 71 citations · Cited by this paper
  28. Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy · Eirini Kyriakopoulou · 2023 · 69 citations · Cited by this paper
  29. AAV9:PKP2 improves heart function and survival in a Pkp2-deficient mouse model of arrhythmogenic right ventricular cardiomyopathy · Iris Wu · 2024 · 55 citations · Cited by this paper
  30. Role of the lectin pathway of complement in hematopoietic stem cell transplantation-associated endothelial injury and thrombotic microangiopathy · Eleni Gavriilaki · 2021 · 44 citations · Cited by this paper
  31. 261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands · Laurent Servais · 2023 · 42 citations · Cited by this paper
  32. Correction of half the cardiomyocytes fully rescue Friedreich ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms · Brahim Belbellaa · 2018 · 40 citations · Cited by this paper
  33. Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74 · Rachael A. Potter · 2024 · 39 citations · Cited by this paper
  34. Seroprevalence of Adeno-Associated Virus Neutralizing Antibodies in Males with Duchenne Muscular Dystrophy · Sumit Verma · 2022 · 30 citations · Cited by this paper
  35. Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy · Barath Jagadisan · 2023 · 30 citations · Cited by this paper
  36. Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy · Jiali Nie · 2023 · 29 citations · Cited by this paper
  37. First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3 -associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1 · Milind Y. Desai · 2025 · 20 citations · Cited by this paper
  38. Real-World Artificial Intelligence–Based Electrocardiographic Analysis to Diagnose Hypertrophic Cardiomyopathy · Milind Y. Desai · 2025 · 19 citations · Cited by this paper
  39. Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery · Rebecca C. Ahrens‐Nicklas · 2026 · 19 citations · Cited by this paper

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