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Human Gene Therapy|December 8, 2023
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67phox-Deficient Chronic Granulomatous DiseaseThomas E Whittaker, Shefta E Moula, Sameer Bahal, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 20, 2005
A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cellsAurelie Olivier, Laurence Jeanson-Leh, Gerben Bouma, et al.
Human Gene Therapy|April 9, 2016
Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type IDiego Leon-Rico, Montserrat Aldea, Raquel Sanchez-Baltasar, et al.
Blood|November 20, 2012
Disease-associated missense mutations in the EVH1 domain disrupt intrinsic WASp function causing dysregulated actin dynamics and impaired dendritic cell migrationAusten J J Worth, Joao Metelo, Gerben Bouma, et al.
Blood|September 20, 2002
Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapyKatie Binley, Zoe Askham, Sharifah Iqball, et al.
The Journal of Gene Medicine|September 2, 2003
Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapyFrank C Schlichtenbrede, Lyndon da Cruz, Clare Stephens, et al.
Gene Therapy|June 24, 2025
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW miceYing Hong, Alice Burleigh, Aiyin Liao, et al.
Experimental Hematology|March 29, 2014
Dose response and clonal variability of lentiviral tetracycline-regulated vectors in murine hematopoietic cellsOlga S Kustikova, Maike Stahlhut, Teng-Cheong Ha, et al.
European Journal of Immunology|October 12, 2013
TCR-engineered T cells: a model of inducible TCR expression to dissect the interrelationship between two TCRsSimone Reuß, Zsolt Sebestyén, Niels Heinz, et al.
Human Gene Therapy|September 10, 2010
Retroviral and transposon-based tet-regulated all-in-one vectors with reduced background expression and improved dynamic rangeNiels Heinz, Axel Schambach, Melanie Galla, et al.
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