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JAMA, ISSN 0098-7484, 04/2015, Volume 313, Issue 15, pp. 1550 - 1563
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 09/2008, Volume 118, Issue 9, pp. 3143 - 3150
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 10/2014, Volume 371, Issue 15, pp. 1407 - 1417
Journal Article
Blood, ISSN 0006-4971, 03/2011, Volume 117, Issue 11, pp. 3243 - 3246
Severe combined immunodeficiency (SCID) carries a poor prognosis without definitive treatment by hematopoietic stem cell transplantation. The outcome for... 
GENE-THERAPY | HEMATOLOGY | RECONSTITUTION | DEFICIENCY | TRANSPLANTATION | Severe Combined Immunodeficiency - diagnosis | Humans | Survival Analysis | Hematopoietic Stem Cell Transplantation | Treatment Outcome | Neonatal Screening | Infant, Newborn | Cohort Studies | Siblings
Journal Article
Blood, ISSN 0006-4971, 11/2008, Volume 112, Issue 9, pp. 3907 - 3913
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 08/2007, Volume 117, Issue 8, pp. 2241 - 2249
Journal Article
JAMA: Journal of the American Medical Association, ISSN 0098-7484, 04/2015, Volume 313, Issue 15, pp. 1550 - 1550
Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen-matched allogeneic... 
Journal Article
Obstetrical & Gynecological Survey, ISSN 0029-7828, 07/2011, Volume 66, Issue 7, pp. 398 - 399
Infants born with severe combined immunodeficiencies (SCIDs) develop severe and recurrent infections in the first few months of life that are fatal without... 
Journal Article
by Nathwani, Amit and Badar, Adam and Rosales, Cecilia and Chokshi, Shilpa and Kia, Azadeh and Nathwani, Devhrut and Galante, Eva and Arstad, Erik and Davidoff, Andrew and Williams, Roger and Lythgoe, Mark and Nathwani, Amit and Le Guiner, Caroline and Cherel, Yan and Montus, Marie and Servais, Laurent and Hogrel, Jean-Yves and Carlier, Pierre and Masurier, Carole and Adjali, Oumeya and Barnay-Toutain, Fréderic and Koo, Taeyoung and Athanasopoulos, Takis and Malerba, Alberto and Mavilio, Fulvio and Moullier, Philippe and Voit, Thomas and Buckland, Karen and Rivat, Christine and Himoudi, Nourredine and Gilmour, Kimberly and Booth, Claire and Xu-Bayford, Jinhua and Cornetta, Kenneth and Kohn, Donald and Carbonaro, Denise and Paruzynski, Anna and Schmidt, Manfred and Thrasher, Adrian and Taher, Taher and Mullen, Lisa and Pastan, Ira and Marshall, John and Chernajovsky, Yuti and Gould, David and Mageed, Rizgar and Carlisle, Robert and Fisher, Kerry and Seymour, Len and Choudhary, Prateek and de Cassan, Simone and Biswas, Sumi and Collins, Katharine and Halstead, Fenella and Bliss, Carly and Ewer, Katie and Sheehy, Susanne and Duncan, Christopher and Hill, Adrian and Draper, Simon and Dakin, Rachel and Parker, Alan and Ma, Jiangtao and Custers, Jerome and Custers, Jerome and Nicklin, Stuart and Baker, Andrew and Meyer, Joel and Harris, Stephanie and Satti, Iman and Sheehan, Sharon and Bettinson, Henry and McShane, Helen and McMutrie, Donna and Sheridan, Sarah and Armstrong, Alison and Ward, Barbara and Callander, Emma and Walsh, Anthony and Weinberg, Benjamin and Chakravarti, Deboki and Wong, Wilson and Bagoanda, Niquita and Ignatova, Svetlana and Fisher, Derek and Themis, Michael and Gould, David and Calero-Garcia, Miguel and Thrasher, Adrian J and Gaspar, H. Bobby and Vlachou, Thalia and Antoniou, Michael and Deng, Lin and White, Katie and Uil, Taco and Kelly, Sharon M and Nicklin, Stuart A and Baker, Andrew H and Howe, Steven and Howe, Steven and ...
Human Gene Therapy, ISSN 1043-0342, 05/2014, Volume 25, Issue 5, pp. A1 - A22
Journal Article
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