2016
DOI: 10.1182/blood-2016-07-729871
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Gene-corrected human Munc13-4–deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice

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Cited by 25 publications
(32 citation statements)
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“…As described previously, 4 we designed a self-inactivating LV carrying a codon-optimized human UNC13D or GFP cDNA under the control of a short elongation factor 1-a (EF1-a) promoter. In a series of independent experiments, Sca1…”
Section: Resultsmentioning
confidence: 99%
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“…As described previously, 4 we designed a self-inactivating LV carrying a codon-optimized human UNC13D or GFP cDNA under the control of a short elongation factor 1-a (EF1-a) promoter. In a series of independent experiments, Sca1…”
Section: Resultsmentioning
confidence: 99%
“…4 If approved by regulatory bodies, the use of gene-corrected autologous T cells would be a treatment option for FHL3 patients. However, it has recently been demonstrated that (1) NK cell cytotoxicity has a distinct immunoregulatory role in HLH disease, 19 and (2) Munc13-4 has a critical function in platelets and neutrophils.…”
Section: Discussionmentioning
confidence: 99%
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“…Recently published preclinical data suggest that lentiviral vector-based T cell gene therapy might be applicable to other inherited and acquired T lymphocyte diseases, such as familial hemophagocytic lymphohistiocytosis types 2 8 and 3 9 immunodysregulation, polyendocrinopathy, enteropathy, X-linked syndrome, 10 and HIV infection 11 . A prerequisite for the efficient achievement of this goal in the clinic is a better understanding of the in vivo cell biology of gene-modified T cells.…”
Section: Introductionmentioning
confidence: 99%