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Journal of Clinical Immunology|August 15, 2013
Combined de-novo mutation and non-random X-chromosome inactivation causing Wiskott-Aldrich syndrome in a female with thrombocytopeniaBoonchai Boonyawat, Santhosh Dhanraj, Fahad Al Abbas, et al.
Stem Cells (Dayton, Ohio)|September 5, 2014
Sheep CD34+ amniotic fluid cells have hematopoietic potential and engraft after autologous in utero transplantationS W Steven Shaw, Michael P Blundell, Caterina Pipino, et al.
The Journal of Experimental Medicine|March 25, 2009
The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and functionMichela Locci, Elena Draghici, Francesco Marangoni, et al.
Science Translational Medicine|August 26, 2011
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correctionH Bobby Gaspar, Samantha Cooray, Kimberly C Gilmour, et al.
Proceedings of the National Academy of Sciences of the United States of America|October 7, 2009
Phosphorylation of WASp is a key regulator of activity and stability in vivoMichael P Blundell, Gerben Bouma, Joao Metelo, et al.
Cellular Reprogramming|August 28, 2014
Trisomy 21 mid-trimester amniotic fluid induced pluripotent stem cells maintain genetic signatures during reprogramming: implications for disease modeling and cryobankingCaterina Pipino, Sayandip Mukherjee, Anna L David, et al.
The New England Journal of Medicine|April 19, 2002
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapySalima Hacein-Bey-Abina, Françoise Le Deist, Frédérique Carlier, et al.
Nature|September 22, 2006
Gene therapy: is IL2RG oncogenic in T-cell development?Karin Pike-Overzet, Dick de Ridder, Floor Weerkamp, et al.
The Journal of Investigative Dermatology|January 15, 2016
Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEBChristos Georgiadis, Farhatullah Syed, Anastasia Petrova, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 22, 2012
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activityClaudia A Montiel-Equihua, Lin Zhang, Sean Knight, et al.
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