Ontology highlight
ABSTRACT:
SUBMITTER: Boztug K
PROVIDER: S-EPMC3064520 | biostudies-literature | 2010 Nov
REPOSITORIES: biostudies-literature
Boztug Kaan K Schmidt Manfred M Schwarzer Adrian A Banerjee Pinaki P PP Díez Inés Avedillo IA Dewey Ricardo A RA Böhm Marie M Nowrouzi Ali A Ball Claudia R CR Glimm Hanno H Naundorf Sonja S Kühlcke Klaus K Blasczyk Rainer R Kondratenko Irina I Maródi László L Orange Jordan S JS von Kalle Christof C Klein Christoph C
The New England journal of medicine 20101101 20
The Wiskott-Aldrich syndrome (WAS) is an X-linked recessive primary immunodeficiency disorder associated with thrombocytopenia, eczema, and autoimmunity. We treated two patients who had this disorder with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC). We found sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B cells, natural killer (NK) cells, and monocytes were functionally corrected. Af ...[more]