Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

• Navigating disruption in the PID landscape: embracing opportunities and anticipating threats in the next ten years

  2025 Frontiers in Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

  2

  citations
• Donor insertion into CX3CR1 allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiation

  2025 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  3

  citations
• Safety and efficacy of gene therapy for RAG1-deficient SCID

  2025 Molecular Therapy article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  2

  citations
• Gene therapy strategies for RAG1 deficiency: Challenges and breakthroughs

  2024 Immunology Letters article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  8

  citations
• Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

  2023 Frontiers in Immunology article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 1 patent

  4

  citations
• Advances in gene therapy for inborn errors of immunity

  2023 Current Opinion in Allergy and Clinical Immunology article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  23

  citations
• The recombinase activating genes: architects of immune diversity during lymphocyte development

  2023 Frontiers in Immunology article Biochemistry, Genetics and Molecular Biology DNA Repair Mechanisms Open access

  32

  citations
• Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

  2021 Molecular Therapy article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 1 patent

  51

  citations
• Multiple Genes Surrounding Bcl-x L , a Common Retroviral Insertion Site, Can Influence Hematopoiesis Individually or in Concert

  2020 Human Gene Therapy article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research

  8

  citations
• Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

  2020 Pharmaceutics article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access

  10

  citations
• A primer to gene therapy: Progress, prospects, and problems

  2020 Journal of Inherited Metabolic Disease article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 4 patents

  27

  citations
• Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

  2020 Molecular Therapy — Methods & Clinical Development article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access cited by 3 patents

  58

  citations

12 results