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

Cited by (264)

Items 101–200 of 264

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9. Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing
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15. Flow Cytometry as a Diagnostic Tool in Primary and Secondary Immune Deficiencies
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16. Integrome signatures of lentiviral gene therapy for SCID-X1 patients
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17. Severe combined immune deficiency
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18. Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side
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19. New perspectives in gene therapy for inherited disorders
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24. Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy
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29. Gene Therapy for Inborn Errors of Immunity
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31. Localising movement disorders in childhood
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33. A Novel Non-Coding Variant in DCLRE1C Results in Deregulated Splicing and Induces SCID Through the Generation of a Truncated ARTEMIS Protein That Fails to Support V(D)J Recombination and DNA Damage Repair
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36. Severe Combined Immunodeficiency: A Guide for Primary Care Givers
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37. Molecular Diagnosis of Inherited Immune Disorders
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39. Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndrome
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41. Severe Combined Immunodeficiency in the Newborn Period
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42. Systematic review of literature and analysis of big data from the National Health Insurance System on primary immunodeficiencies in Korea
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