ArticleGene therapy2003
Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein.
Article in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 58 citations in OpenAlex.
- Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- WASp Deficiency Selectively Affects the TCR Diversity of Different Memory T Cell Subsets in WAS Chimeric Mice.Frontiers in immunology · 2021Article
- Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.Nature communications · 2016Article
- Clinical applications of gene therapy for primary immunodeficiencies.Human gene therapy · 2015Review
- Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.Human gene therapy. Clinical development · 2013Article
- IL-2 induces a WAVE2-dependent pathway for actin reorganization that enables WASp-independent human NK cell function.The Journal of clinical investigation · 2011Article
- Transduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteins.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Wiskott-Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation.Immunobiology · 2009Review
- Development of lentiviral gene therapy for Wiskott Aldrich syndrome.Expert opinion on biological therapy · 2008Review
- Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.Gene therapy · 2007Article
- WIP is a chaperone for Wiskott-Aldrich syndrome protein (WASP).Proceedings of the National Academy of Sciences of the United States of America · 2007Article
- Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation.Bone marrow transplantation · 2006Article
- Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences.Gene therapy · 2005Article
- Lentiviral vectors for the treatment of Wiskott-Aldrich syndrome.Gene therapy · 2005Article
- A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.Gene therapy · 2005Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
T-cell dysfunction is thought to be central to the immunodeficiency state seen in patients with the Wiskott-Aldrich syndrome (WAS). Aspects of the WAS phenotype have been corrected in other cell types on introduction of the normal WAS protein (WASP), but the potential for correction of the T-cell defects has not been evaluated. Here we demonstrate that an oncoretroviral vector encoding WASP and green fluorescent protein (GFP), and pseudotyped with the RD114 envelope protein, efficiently transduces primary human T cells derived from WAS patients. Transcription initiated at the oncoretroviral long terminal repeat (LTR) results in levels of WASP that, while lower than those seen in normal control T cells, resulted in correction of the deficient proliferative response to T-cell receptor (TCR) stimulation characteristic of WAS. IL2 secretion after TCR stimulation was partially corrected. Control primary T cells transduced with the same vector responded normally to TCR stimulation, and showed no increase in WASP expression. The demonstration that correction of T cell defects can be achieved by gene transfer supports continued efforts to develop gene therapy for WAS.
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Registered trials
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