Research map: Gene therapy approaches for inborn errors of immunity: from bench to bedside

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Papers in this map

  1. LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 · S. Hacein-Bey-Abina · 2003 · 3601 citations · Cited by this paper
  2. Aflibercept-ayyh (aflibercept 2 mg biosimilar) for treatment-naïve retinal vascular diseases: early real-world experience – the APEX study · 2026 · Related
  3. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 · Salima Hacein‐Bey‐Abina · 2008 · 1864 citations · Cited by this paper
  4. Efficacy of intravenous infliximab in axial spondyloarthritis: impact on disease activity, quality of life, and productivity in subgroups with psoriasis and inflammatory bowel disease · 2026 · Related
  5. T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years · R. Michael Blaese · 1995 · 1552 citations · Cited by this paper
  6. Twelve-month changes in lipid-derived cardiovascular risk indices among patients with psoriasis receiving biologic therapy: a multicenter real-world retrospective cohort study · 2026 · Related
  7. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients · Steven J. Howe · 2008 · 1300 citations · Cited by this paper
  8. Anakinra treatment practices in colchicine-resistant familial Mediterranean fever: a survey of pediatric rheumatologists conducted through the PReS AID-WP and the Turkish pediatric rheumatology associations · 2026 · Related
  9. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia · Marina Cavazzana · 2010 · 1288 citations · Cited by this paper
  10. Immunotherapy for patients with melanoma in East Asia: current evidence, future directions, and clinical implications · 2026 · Related
  11. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 · Marion Gabriele Ott · 2006 · 1196 citations · Cited by this paper
  12. Targeting the neuroimmune axis in prurigo nodularis: a critical appraisal of the nemolizumab trials · 2026 · Related
  13. Human Inborn Errors of Immunity: 2022 Update on the Classification from the International Union of Immunological Societies Expert Committee · Stuart G. Tangye · 2022 · 1192 citations · Cited by this paper
  14. The role of monoclonal antibodies in the treatment of lupus nephritis · 2026 · Related
  15. Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome · Alessandro Aiuti · 2013 · 1041 citations · Cited by this paper
  16. Identification of plaque psoriasis subgroups based on peripheral blood immune cells subtypes and their relationship with biologic therapy efficacy: a single-center longitudinal cohort study in China · 2026 · Related
  17. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease · STEFAN P. STEIN · 2010 · 787 citations · Cited by this paper
  18. Etuvetidigene Autotemcel: First Approval · 2026 · Related
  19. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector · Hubert Bobby Gaspar · 2004 · 689 citations · Cited by this paper
  20. Regional disparities in access to gene therapies in the European Union, the United States, Japan, and China · 2025 · Related
  21. Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity · Christian Joerg Braun · 2014 · 562 citations · Cited by this paper
  22. A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency · Salima Hacein‐Bey‐Abina · 2014 · 414 citations · Cited by this paper
  23. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease · Kiran Musunuru · 2025 · 357 citations · Cited by this paper
  24. Human inborn errors of immunity: 2024 update on the classification from the International Union of Immunological Societies Expert Committee · M. Cecilia Poli · 2025 · 333 citations · Cited by this paper
  25. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study · Federica Barzaghi · 2017 · 320 citations · Cited by this paper
  26. Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study · Jennifer R. Heimall · 2017 · 279 citations · Cited by this paper
  27. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1 · Ewelina K. Mamcarz · 2019 · 271 citations · Cited by this paper
  28. Lentiviral gene therapy for X-linked chronic granulomatous disease · Donald B. Kohn · 2020 · 265 citations · Cited by this paper
  29. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency · Donald B. Kohn · 2021 · 225 citations · Cited by this paper
  30. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study · Francesca Ferrua · 2019 · 213 citations · Cited by this paper
  31. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges · Marina Cavazzana · 2019 · 202 citations · Cited by this paper
  32. Gene correction for SCID-X1 in long-term hematopoietic stem cells · Mara Pavel-Dinu · 2019 · 197 citations · Cited by this paper
  33. Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease · Suk See De Ravin · 2016 · 195 citations · Cited by this paper
  34. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome · Caroline Y. Kuo · 2018 · 142 citations · Cited by this paper
  35. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy · Christine Duncan · 2024 · 142 citations · Cited by this paper
  36. Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome · Rajeev Rai · 2020 · 133 citations · Cited by this paper
  37. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome · Alessandra Magnani · 2022 · 123 citations · Cited by this paper
  38. CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells · Marianne Goodwin · 2020 · 120 citations · Cited by this paper
  39. Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug · Francesca Ferrua · 2017 · 103 citations · Cited by this paper

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