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

• Published in: The New England journal of medicine Vol. 380; no. 16; pp. 1525 - 1534
• Main Authors: Mamcarz, Ewelina, Zhou, Sheng, Lockey, Timothy, Abdelsamed, Hossam, Cross, Shane J, Kang, Guolian, Ma, Zhijun, Condori, Jose, Dowdy, Jola, Triplett, Brandon, Li, Chen, Maron, Gabriela, Aldave Becerra, Juan C, Church, Joseph A, Dokmeci, Elif, Love, James T, da Matta Ain, Ana C, van der Watt, Hedi, Tang, Xing, Janssen, William, Ryu, Byoung Y, De Ravin, Suk See, Weiss, Mitchell J, Youngblood, Benjamin, Long-Boyle, Janel R, Gottschalk, Stephen, Meagher, Michael M, Malech, Harry L, Puck, Jennifer M, Cowan, Morton J, Sorrentino, Brian P
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 18.04.2019
• Subjects: Antigens, Differentiation, T-Lymphocyte - blood; Autografts; B-Lymphocytes - physiology; Babies; Bone marrow; Busulfan; Busulfan - administration & dosage; CD3 antigen; CD4 antigen; Chemotherapy; Complementary DNA; Expression vectors; Gene therapy; Genetic Therapy; Genetic Vectors; Globulins; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Hospitals; Humans; Immunoglobulin M; Immunoglobulin M - blood; Infant; Infants; Interleukin Receptor Common gamma Subunit - genetics; Intravenous administration; Killer Cells, Natural; Laboratories; Lentivirus; Lymphocyte Count; Lymphocytes B; Lymphocytes T; Male; Medical screening; Myeloid cells; Natural killer cells; Osteoprogenitor cells; Pediatrics; Severe combined immunodeficiency; Stem cell transplantation; Stem cells; Supplements; T-Lymphocytes; Transplantation Conditioning; Transplants & implants; X-Linked Combined Immunodeficiency Diseases - genetics; X-Linked Combined Immunodeficiency Diseases - immunology; X-Linked Combined Immunodeficiency Diseases - therapy; United States > US; San Francisco California; California
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMoa1815408

Eight infants with SCID-X1 had multilineage immune reconstitution after autologous hematopoietic stem-cell transplantation of marrow stem cells transfected with a lentiviral vector containing IL2RG cDNA after busulfan conditioning. Previous infections cleared and functional T cells developed in all eight infants, IgM levels normalized in seven, and three had a response to vaccines.