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Molecular Therapy : the Journal of the American Society of Gene Therapy|August 9, 2005
Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal miceMike Themis, Simon N Waddington, Manfred Schmidt, et al.
Lancet (London, England)|December 22, 2004
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vectorH Bobby Gaspar, Kathryn L Parsley, Steven Howe, et al.
Human Mutation|July 24, 2010
A custom 148 gene-based resequencing chip and the SNP explorer software: new tools to study antibody deficiencyHong-Ying Wang, Vivek Gopalan, Ivona Aksentijevich, et al.
Journal of Clinical Immunology|May 28, 2013
A novel gain-of-function IKBA mutation underlies ectodermal dysplasia with immunodeficiency and polyendocrinopathyLena F Schimke, Nikolaus Rieber, Stacey Rylaarsdam, et al.
Gastroenterology|June 12, 2010
Reduced expression of FOXP3 and regulatory T-cell function in severe forms of early-onset autoimmune enteropathyNicolette Moes, Frédéric Rieux-Laucat, Bernadette Begue, et al.
Stem Cells (Dayton, Ohio)|May 23, 2019
In Utero Transplantation of Expanded Autologous Amniotic Fluid Stem Cells Results in Long-Term Hematopoietic EngraftmentStavros P Loukogeorgakis, Panicos Shangaris, Enrica Bertin, et al.
Science Translational Medicine|January 27, 2017
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cellsWaseem Qasim, Hong Zhan, Sujith Samarasinghe, et al.
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.
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