scholarly journals Towards gene therapy for EBV-associated posttransplant lymphoma with genetically modified EBV-specific cytotoxic T cells

Blood ◽  
2014 ◽  
Vol 124 (16) ◽  
pp. 2514-2522 ◽  
Author(s):  
Ida Ricciardelli ◽  
Michael Patrick Blundell ◽  
Jennifer Brewin ◽  
Adrian Thrasher ◽  
Martin Pule ◽  
...  

Key Points EBV-CTLs resistant to calcineurin inhibitors mediate durable, potent antitumor responses despite immunosuppression in a murine model of PTLD. This approach improves immunotherapy efficacy with EBV-CTLs for PTLD after SOT and obviates need for immunosuppression withdrawal.

Blood ◽  
2013 ◽  
Vol 121 (12) ◽  
pp. 2224-2233 ◽  
Author(s):  
Ashley T. Martino ◽  
Etiena Basner-Tschakarjan ◽  
David M. Markusic ◽  
Jonathan D. Finn ◽  
Christian Hinderer ◽  
...  

Key Points A murine model was developed for capsid-specific CD8 cell responses in AAV gene therapy for hemophilia. Y-F mutant capsid minimizes the effect of anticapsid CD8+ T cells on hepatocyte-derived factor IX expression in mice and in human cells.


2002 ◽  
Vol 168 (10) ◽  
pp. 5376.2-5376
Author(s):  
Larisa Y. Poluektova ◽  
David H. Munn ◽  
Yuri Persidsky ◽  
Howard E. Gendelman

2008 ◽  
Vol 2 ◽  
pp. CMO.S586
Author(s):  
Herrero Mj ◽  
R Botella ◽  
R Algás ◽  
FM Marco ◽  
Aliño Sf

Cancer vaccines have always been in the scope of gene therapy research. One of the most successful approaches has been working with genetically modified tumor cells. However, to become a clinical reality, tumor cells must suffer a long and risky process from the extraction from the patient to the reimplantation as a vaccine. In this work, we explain our group's approach to reduce the cell number required to achieve an immune response against a melanoma murine model, employing bead-selected B16 tumor cells expressing GM-CSF and B7.2.


Blood ◽  
2015 ◽  
Vol 126 (26) ◽  
pp. 2882-2891 ◽  
Author(s):  
Prashant Hiwarkar ◽  
Waseem Qasim ◽  
Ida Ricciardelli ◽  
Kimberly Gilmour ◽  
Sergio Quezada ◽  
...  

Key Points CB T cells mediate enhanced antitumor responses compared with PB T cells in a murine model of B-cell lymphoma. The antitumor activity correlates with increased tumor-homing of CCR7high CB CD8+ T cells and rapid gain of cytotoxic and Th1 function.


Hematology ◽  
2009 ◽  
Vol 2009 (1) ◽  
pp. 675-681 ◽  
Author(s):  
Malcolm K. Brenner ◽  
Fatma V. Okur

Abstract It is now twenty years since the first legal gene transfer studies were approved, and there has been considerable disappointment in the slow rate of progress that followed the initial studies. Gradually, however, as the limitations of available vectors are acknowledged and overcome, and with advances in our understanding of the molecular and cell biology of genetic diseases and of cancer, unequivocal successes are now being reported. In this paper we describe the remaining major roadblocks to successful gene therapy and outline approaches to overcome them. We also illustrate how genetically modified immune system cells are already being used for the effective treatment of hematological and other malignancies, and how these approaches are being modified so that they can be effective in treating a broader range of malignancies.


2017 ◽  
Vol 1 (27) ◽  
pp. 2781-2789 ◽  
Author(s):  
Tayebeh Soheili ◽  
Amandine Durand ◽  
Fernando E. Sepulveda ◽  
Julie Rivière ◽  
Chantal Lagresle-Peyrou ◽  
...  

Key Points UNC13D gene transfer into HSCs corrects all clinical and biological signs of HLH in a mouse model. Munc13-4 is expressed in mature CD8+ T cells allowing the correction of cytotoxic activity and consequently efficient viral restriction.


Blood ◽  
2017 ◽  
Vol 130 (2) ◽  
pp. 146-155 ◽  
Author(s):  
David M. Woods ◽  
Karrune V. Woan ◽  
Fengdong Cheng ◽  
Andressa L. Sodré ◽  
Dapeng Wang ◽  
...  

Key Points T cells from HDAC11KO mice have increased effector functions and mediate more rapid and potent GVHD. HDAC11 associates with the Eomes and Tbet gene promoter regions in resting cells and disassociates upon activation.


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