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Molecular Therapy : the Journal of the American Society of Gene Therapy|November 22, 2013
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiencyDenise A Carbonaro, Lin Zhang, Xiangyang Jin, et al.Human Gene Therapy|March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|March 8, 2013
Outcome of transplantation for acute myelogenous leukemia in children with Down syndromeJohann K Hitzler, Wensheng He, John Doyle, et al.Pediatric Blood & Cancer|January 7, 2014
Outcome of transplantation for acute lymphoblastic leukemia in children with Down syndromeJohann K Hitzler, Wensheng He, John Doyle, et al.Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology|January 12, 2011
Reducing the risk for transplantation-related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made?John T Horan, Brent R Logan, Manza-A Agovi-Johnson, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 15, 2022
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trialsKenneth Cornetta, Jing Yao, Kimberley House, et al.The New England Journal of Medicine|October 15, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase DeficiencyClaire Booth, Katelyn Masiuk, Konstantinos Vazouras, et al.Nature|September 17, 2010
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaMarina Cavazzana-Calvo, Emmanuel Payen, Olivier Negre, et al.Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|March 18, 2014
Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanomaThinle Chodon, Begoña Comin-Anduix, Bartosz Chmielowski, et al.Blood|May 11, 2021
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiencyBryanna Reinhardt, Omar Habib, Kit L Shaw, et al.Pageof 15