Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
Open accessAbstract
BACKGROUND: Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy is given. In previous studies, autologous gene therapy with γ-retroviral vectors failed to reconstitute B-cell and NK-cell immunity and was complicated by vector-related leukemia. METHODS: complementary DNA to bone marrow stem cells after low-exposure, targeted busulfan conditioning in eight infants with newly diagnosed SCID-X1. RESULTS: Eight infants with SCID-X1 were followed for a median of 16.4 months. Bone marrow harvest, busulfan conditioning, and cell infusion had no unexpected side effects. In seven infants, the numbers of CD3+, CD4+, and naive CD4+ T cells and NK cells normalized by 3 to 4 months after infusion and were accompanied by vector marking in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors. The eighth infant had an insufficient T-cell count initially, but T cells developed in this infant after a boost of gene-corrected cells without busulfan conditioning. Previous infections cleared in all infants, and all continued to grow normally. IgM levels normalized in seven of the eight infants, of whom four discontinued intravenous immune globulin supplementation; three of these four infants had a response to vaccines. Vector insertion-site analysis was performed in seven infants and showed polyclonal patterns without clonal dominance in all seven. CONCLUSIONS: Lentiviral vector gene therapy combined with low-exposure, targeted busulfan conditioning in infants with newly diagnosed SCID-X1 had low-grade acute toxic effects and resulted in multilineage engraftment of transduced cells, reconstitution of functional T cells and B cells, and normalization of NK-cell counts during a median follow-up of 16 months. (Funded by the American Lebanese Syrian Associated Charities and others; LVXSCID-ND ClinicalTrials.gov number, NCT01512888.).
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Lentiviral vectors recently reached the clinic for the treatment of a severe combined immunodeficiency.
“Lentiviral vectors recently reached the clinic for the treatment of a severe combined immunodeficiency56.”
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Lentiviral Vector Production from a Stable Packaging Cell Line Using a Packed Bed Bioreactor
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A Review of Gene Therapy Delivery Systems for Intervertebral Disc Degeneration
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Evaluating the probability of CRISPR‐based gene drive contaminating another species
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The Treatment of Primary Immune Deficiencies: Lessons Learned and Future Opportunities
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Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia
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A primer to gene therapy: Progress, prospects, and problems
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CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside
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Gene therapy for X-linked severe combined immunodeficiency: Historical outcomes and current status
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The transformative potential of HSC gene therapy as a genetic medicine
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Development and clinical translation of ex vivo gene therapy
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In-Vivo Gene Therapy with Foamy Virus Vectors
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Transcriptomic analysis of the innate immune response to in vitro transfection of plasmid DNA
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Gene therapy for primary immunodeficiencies
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Treatment of patients with immunodeficiency: Medication, gene therapy, and transplantation
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Genomics and epigenetics guided identification of tissue-specific genomic safe harbors
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Self-citationIn Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations
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- Authors
- Mamcarz, E., Zhou, S., Lockey, T., Abdelsamed, H., Cross, S. J., Kang, G., Ma, Z., Condori, J., Dowdy, J., Triplett, B., Li, C., Maron, G., Aldave Becerra, J. C., Church, J. A., Dokmeci, E., Love, J. T., da Matta Ain, A. C., van der Watt, H., Tang, X., Janssen, W., Ryu, B. Y., De Ravin, S. S., Weiss, M. J., Youngblood, B., Long-Boyle, J. R., Gottschalk, S., Meagher, M. M., Malech, H. L., Puck, J. M., Cowan, M. J., & Sorrentino, B. P.
- Affiliations
- St. Jude Children's Research Hospital (US)
- From the Departments of Bone Marrow Transplantation and Cellular Therapy (E.M., B.T., W.J., S.G.), Hematology (S.Z., Z.M., J.C., J.D., X.T., B.Y.R., M.J.W., B.P.S.), Therapeutics Production and Quality (T.L., M.M.M.), Immunology (H.A., B.Y.), Pharmaceutical Sciences (S.J.C.), Biostatistics (G.K., C.L.), and Infectious Diseases (G.M.), St. Jude Children’s Research Hospital, Memphis, TN; the Allergy and Clinical Immunology Division, Hospital Nacional Edgardo Rebagliati Martins, Lima, Peru (J.C.A.B.);...
- Children's Hospital Los Angeles (US)
- University of New Mexico (US)
- University of Oklahoma Health Sciences Center (US)
- National Institutes of Health (US)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2019-04-18
- Volume
- 380
- Issue
- 16
- Pages
- 1525-1534
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- PMC
- PMC6636624
- OpenAlex
- W2937292311
- Cited by
- 263
- References
- 32
- Citations by year
- 2026: 11 · 2025: 18 · 2024: 30 · 2023: 36 · 2022: 42 · 2021: 53 · 2020: 46 · 2019: 26 · 2018: 1
- FWCI
- 25.16
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- LA Referencia (Red Federada de Repositorios Institucionales de Publicaciones Científicas) (publishedVersion)
- PubMed Central (acceptedVersion)
- UNM’s Digital Repository (University of New Mexico) (submittedVersion)
- Americanae (AECID Library) (submittedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- California Institute of Regenerative Medicine (CLIN2-09504)
- National Heart, Lung, and Blood Institute (P01 HL053749)
- Assisi Foundation of Memphis
- Division of Intramural Research, National Institute of Allergy and Infectious Diseases (U54-AI-082973, Z01-A1-00988)
- American Lebanese Syrian Associated Charities
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Immunology and Microbiology › Immunology › Immunodeficiency and Autoimmune Disorders
- Life Sciences › Immunology and Microbiology › Virology › HIV Research and Treatment
- Keywords
- X-linked severe combined immunodeficiency, busulfan conditioning, autologous gene therapy, bone marrow-derived stem cells, T cell reconstitution, B cell reconstitution, NK cell reconstitution, intravenous immunoglobulin, vaccine response, infants
- MeSH terms
- Antigens, Differentiation, T-Lymphocyte, B-Lymphocytes, Busulfan, Genetic Vectors, Humans, Immunoglobulin M, Infant, Killer Cells, Natural, Male, T-Lymphocytes, Genetic Therapy, Lentivirus, Hematopoietic Stem Cell Transplantation, Lymphocyte Count, Transplantation Conditioning, Interleukin Receptor Common gamma Subunit, X-Linked Combined Immunodeficiency Diseases
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