Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.

Autor: Labrosse R; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.; Division of Allergy and Immunology, Department of Pediatrics, CHU Sainte-Justine, University of Montreal, Montreal, QC, Canada., Chu JI; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Division of Pediatric Allergy, Immunology and Bone Marrow Transplantation, Benioff Children's Hospital, University of California San Francisco, San Francisco, CA., Armant MA; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Pellin D; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Kareddy N; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Frelinger AL; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Henderson LA; Division of Immunology, Boston Children's Hospital, Boston, MA., O'Connell AE; Division of Newborn Medicine, Boston Children's Hospital, Boston, MA., Biswas A; Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Harvard Medical School, Boston, MA., Coenen-van der Spek J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Miggelbrink A; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Fiorini C; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Adhikari H; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Berry CC; Division of Biostatistics and Bioinformatics, Herbert Wertheim School of Public Health, UC San Diego, La Jolla, CA., Cantu VA; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Fong J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Jaroslavsky J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Karadeniz DF; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Li QZ; Department of Immunology, Microarray and Immune Phenotyping Core Facility, University of Texas Southwestern Medical Center, Dallas, TX., Reddy S; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Roche AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Zhu C; Department of Immunology, Microarray and Immune Phenotyping Core Facility, University of Texas Southwestern Medical Center, Dallas, TX., Whangbo JS; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Dansereau C; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Mackinnon B; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Morris E; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Koo SM; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., London WB; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Baris S; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Ozen A; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Karakoc-Aydiner E; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Despotovic JM; Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, TX., Forbes Satter LR; Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, TX., Saitoh A; Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan., Aizawa Y; Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan., King A; Hospital Luis Calvo Mackenna, Clínica Alemana De Santiago Universidad del Desarrollo, Santiago, Chile., Nguyen MAT; Department of Pediatrics, University of Medicine and Pharmacy at Ho Chi Minh City, Ho Chi Minh City, Vietnam., Vu VDU; Department of Pediatrics, University of Medicine and Pharmacy at Ho Chi Minh City, Ho Chi Minh City, Vietnam., Snapper SB; Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Harvard Medical School, Boston, MA., Galy A; Genethon, Évry, France.; University of Paris-Saclay, University of Évry, INSERM, Genethon, Integrare Research Unit UMR_S951, Évry, France., Notarangelo LD; Division of Immunology, Boston Children's Hospital, Boston, MA.; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Williams DA; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Pai SY; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.

Publikováno v: Blood [Blood] 2023 Oct 12; Vol. 142 (15), pp. 1281-1296.