Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Mamcarz, Ewelina, Zhou, Sheng, Lockey, Timothy, Abdelsamed, Hossam, Cross, Shane J., Kang, Guolian, Ma, Zhijun, Condori, Jose +23 more·2019·New England Journal of Medicine
DOI 10.1056/nejmoa1815408

Cited by (264)

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  4. Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation
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  5. Pediatric Bone Marrow Transplantation
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  9. [Advances in newborn screening and immune system reconstitution of severe combined immunodeficiency].
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  10. Gene therapy in radiation oncology
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  18. Pediatric immune deficiencies: current treatment approaches
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  19. Gene Therapy Vectors
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  20. Genetic Engineering
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  21. The Titer of the Lentiviral Vector Encoding Chimeric TRIM5α-HRH Gene is Reduced Due to Expression of TRIM5α-HRH in Producer Cells and the Negative Effect of Ef1α Promoter
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  22. Pediatric Bone Marrow Transplantation
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  23. Updates on Severe Combined Immunodeficiency
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  24. A minimal dose of unmanipulated bone marrow infusion without conditioning for T-B+NK- severe combined immunodeficiency
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  25. New Gene Therapy Potential Cure for “Bubble Boy Disease”: An experimental gene therapy has allowed children with SCID‐1X to develop fully functioning immune systems
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  26. Gene therapy for primary immune deficiencies
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  27. Lentiviral Vector Gene Therapy for X-linked SCID
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  28. Gene Therapy Vectors
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  57. Additional file 10 of Genomics and epigenetics guided identification of tissue-specific genomic safe harbors
    Dewan Shrestha, Aishee Bag, Ruiqiong Wu, Zhang, Yeting, Xing Tang, Qian Qi, Xing, Jinchuan, Yong Cheng · 2022 · Figshare
  58. Gene therapy
    Марахонов Андрей Владимирович, Смирнихина Светлана Анатольевна, Sergey Nikitin, T. Adyan, Maria Akhkiamova, Artem O. Borovikov, Tat'yana Buharova, Irina E. Efimova, Pavel Zharkov, Vitaliy V. Kadyshev et al. · 2026
  59. L'acceptabilité des biotechnologies médicales : les cas spécifiques de la thérapie génique et de la xénotransplantation
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  60. Clinical and genetic landscape of SCID in Yunnan, China: identification of two novel RAG2 variants
    Yanjun Wang, Ruohong Jin, Qian Han, Ling Hang, Ling Lv, Guizhi Chen, Rong Hu, Shufang Xiao · 2026 · Scientific Reports
  61. Gene Therapy As A Treatment Modality For Severe Combined Immunodeficiency : A Narrative Review
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  62. Gene therapy approaches for inborn errors of immunity: from bench to bedside
    Jasmeen Dara, Claire Booth · 2026 · Expert Opinion on Biological Therapy
  63. Evolution of Inborn Errors of Immunity in China: Discoveries and Treatment Paradigms
    Yu Shi, Christopher Chang, Kai Guan · 2026 · Clinical Reviews in Allergy & Immunology
  64. Making space for cures: Conditioning for autologous hematopoietic stem cell gene therapies
    Zachary David Crees, John F. DiPersio, Stephen P. Persaud · 2026 · Seminars in Hematology