Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Alessandro Aiuti, Federica Cattaneo, Stefania Galimberti et al. · New England Journal of Medicine · 2009 · 1K citations · Full text

Medicine, Hematology, Immunology +16

Transplantation Outcomes for Severe Combined Immunodeficiency, 2000–2009

Sung‐Yun Pai, Brent R. Logan, Linda M. Griffith et al. · New England Journal of Medicine · 2014 · 771 citations · Full text

Medicine, Hematology, Pediatrics +14

Targeted genome editing in human repopulating haematopoietic stem cells

Pietro Genovese, Giulia Schiroli, Giulia Escobar et al. · Nature · 2014 · 565 citations · Full text

Life Sciences, Genetics, Hematology +14

Partial V(D)J Recombination Activity Leads to Omenn Syndrome

Anna Villa, Sandro Santagata, Fabio Bozzi et al. · Cell · 1998 · 462 citations · Full text

Life Sciences, Genetics, Molecular Genetics +11

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?

Andrew R. Gennery, Mary Slatter, Laure Grandin et al. · Journal of Allergy and Clinical Immunology · 2010 · 430 citations

Medicine, Hematology, Immunology +16