Research Topics
Genomes and Genes | Richard A MorganSummaryAffiliation: National Institutes of Health Country: USA Publications
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Publications
Both CD4 and CD8 T cells mediate equally effective in vivo tumor treatment when engineered with a highly avid TCR targeting tyrosinaseTimothy L Frankel
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 184:5988-98. 2010..These results suggest that this highly active tyrosinase-specific TCR could be of value in adoptive cell transfer for melanoma...
Recognition of glioma stem cells by genetically modified T cells targeting EGFRvIII and development of adoptive cell therapy for gliomaRichard A Morgan
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 23:1043-53. 2012..Based on these observations, a γ-retroviral vector expressing this EGFRvIII CAR was produced for clinical application...
Cancer regression and neurological toxicity following anti-MAGE-A3 TCR gene therapyRichard A Morgan
Surgery Branch, National Cancer Institute, Bethesda, MD 20892, USA
J Immunother 36:133-51. 2013....
Adoptive cell therapy: genetic modification to redirect effector cell specificityRichard A Morgan
Surgery Branch, National Cancer Institute, 10 Center Drive, Bethesda, MD 20892, USA
Cancer J 16:336-41. 2010..Initial clinical trial results have demonstrated that both T-cell receptor- and chimeric antigen receptor-engineered T cells can be administered to cancer patients and mediate tumor regression...
Case report of a serious adverse event following the administration of T cells transduced with a chimeric antigen receptor recognizing ERBB2Richard A Morgan
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Mol Ther 18:843-51. 2010..We speculate that the large number of administered cells localized to the lung immediately following infusion and were triggered to release cytokine by the recognition of low levels of ERBB2 on lung epithelial cells...
High efficiency TCR gene transfer into primary human lymphocytes affords avid recognition of melanoma tumor antigen glycoprotein 100 and does not alter the recognition of autologous melanoma antigensRichard A Morgan
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 171:3287-95. 2003..These results suggest that lymphocytes genetically engineered to express anti-gp100 TCR may be of value in the adoptive immunotherapy of patients with melanoma...
A high molecular weight melanoma-associated antigen-specific chimeric antigen receptor redirects lymphocytes to target human melanomasWilliam R Burns
Surgery Branch, National Cancer Institute, NIH, Bethesda, Maryland 20892, USA
Cancer Res 70:3027-33. 2010..This may represent a novel means to treat the majority of patients with advanced melanoma, most notably those unable to receive current ACT therapies...
Cancer regression in patients after transfer of genetically engineered lymphocytesRichard A Morgan
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
Science 314:126-9. 2006..This study suggests the therapeutic potential of genetically engineered cells for the biologic therapy of cancer...
Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigenLaura A Johnson
Surgery Branch, Hatfield Clinical Research Center, National Cancer Institute NIH, Bethesda, MD 20892, USA
Blood 114:535-46. 2009..This trial was registered at www.ClinicalTrials.gov as NCI-07-C-0174 and NCI-07-C-0175...
A herceptin-based chimeric antigen receptor with modified signaling domains leads to enhanced survival of transduced T lymphocytes and antitumor activityYangbing Zhao
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 183:5563-74. 2009..More importantly, PBLs expressing this new version of the 4D5 CAR could not only efficiently lyse the autologous fresh tumor digests, but they could strongly suppress tumor growth in a xenogenic mouse model...
T-cell receptor gene therapy of established tumors in a murine melanoma modelJohn D Abad
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunother 31:1-6. 2008..This model may be a powerful tool for evaluating future TCR gene transfer-based strategies...
Presentation of tumor antigens by dendritic cells genetically modified with viral and nonviral vectorsMichal Lotem
Surgery Branch, National Cancer Institute, Bethesda, MD 20892, USA
J Immunother (1997) 29:616-27. 2006..These data suggest that DCs transduced with viral vectors may be more efficient than DCs transfected with cDNAs or RNAs for the induction of tumor reactive CD8+ and CD4+ T cells in vitro and in human vaccination trials...
Human effector CD8+ T cells derived from naive rather than memory subsets possess superior traits for adoptive immunotherapyChristian S Hinrichs
National Cancer Institute, Bethesda, MD, USA
Blood 117:808-14. 2011..Thus, these data suggest that naive cells resist terminal differentiation, or "exhaustion," maintain high replicative potential, and therefore may be the superior subset for use in adoptive immunotherapy...
A simplified method for the clinical-scale generation of central memory-like CD8+ T cells after transduction with lentiviral vectors encoding antitumor antigen T-cell receptorsShicheng Yang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunother 33:648-58. 2010..The methodology described here could be readily applied for engineering CD8+ T cells with antitumor specificity for human adoptive immunotherapy...
Recognition of NY-ESO-1+ tumor cells by engineered lymphocytes is enhanced by improved vector design and epigenetic modulation of tumor antigen expressionJennifer A Wargo
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Cancer Immunol Immunother 58:383-94. 2009..These results have relevance for TCR-based gene therapies targeting common epithelial malignancies...
Transfer of a TCR gene derived from a patient with a marked antitumor response conveys highly active T-cell effector functionsMarybeth S Hughes
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 16:457-72. 2005..TCR-transduced T-cells are thus attractive candidates for evaluation in cell transfer therapies of patients with cancer...
Gene transfer of tumor-reactive TCR confers both high avidity and tumor reactivity to nonreactive peripheral blood mononuclear cells and tumor-infiltrating lymphocytesLaura A Johnson
Surgery Branch, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
J Immunol 177:6548-59. 2006..We propose that inducing expression of this highly avid TCR in patient PBMC has the potential to induce tumor regression, as an "off-the-shelf" reagent for allogeneic melanoma patient gene therapy...
Recognition of fresh human tumor by human peripheral blood lymphocytes transduced with a bicistronic retroviral vector encoding a murine anti-p53 TCRCyrille J Cohen
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 175:5799-808. 2005..These results suggest that lymphocytes genetically engineered to express anti-p53 TCR may be of value for the adoptive immunotherapy of patients with a variety of common malignancies...
Gene therapy using genetically modified lymphocytes targeting VEGFR-2 inhibits the growth of vascularized syngenic tumors in miceDhanalakshmi Chinnasamy
Surgery Branch, National Cancer Institute, Clinical Research Center, Bethesda, Maryland 20892, USA
J Clin Invest 120:3953-68. 2010..T cells transduced with VEGFR-2 CAR showed durable and increased tumor infiltration, correlating with their antitumor effect. This approach provides a potential method for the gene therapy of a variety of human cancers...
Construction and preclinical evaluation of an anti-CD19 chimeric antigen receptorJames N Kochenderfer
Surgery Branch of the National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunother 32:689-702. 2009....
Extrathymic generation of tumor-specific T cells from genetically engineered human hematopoietic stem cells via Notch signalingYangbing Zhao
Surgery Branch, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, Maryland 20892 1201, USA
Cancer Res 67:2425-9. 2007..The genetic manipulation of HSCs has broad implications for ACT of cancer...
Single and dual amino acid substitutions in TCR CDRs can enhance antigen-specific T cell functionsPaul F Robbins
Surgery Branch, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
J Immunol 180:6116-31. 2008....
In vitro generated anti-tumor T lymphocytes exhibit distinct subsets mimicking in vivo antigen-experienced cellsShicheng Yang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
Cancer Immunol Immunother 60:739-49. 2011....
Tumor-specific CD8+ T cells expressing interleukin-12 eradicate established cancers in lymphodepleted hostsSid P Kerkar
Center for Cancer Research, National Cancer Institute, NIH, Bethesda, MD, USA
Cancer Res 70:6725-34. 2010..Our findings reveal an approach to genetically modify T cells to reduce the cell number needed, eliminate the need for vaccines or systemic IL-2, and improve immunotherapy efficacy based on adoptive transfer of gene-engineered T cells...
Adoptive cell therapy for patients with melanoma, using tumor-infiltrating lymphocytes genetically engineered to secrete interleukin-2Bianca Heemskerk
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 19:496-510. 2008..In this study, insertion of the IL-2 gene into antitumor TILs increased their ability to survive after IL-2 withdrawal in vitro but did not increase their in vivo persistence or clinical effectiveness...
Relationship of p53 overexpression on cancers and recognition by anti-p53 T cell receptor-transduced T cellsMarc R Theoret
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892 1201, USA
Hum Gene Ther 19:1219-32. 2008..These studies raise doubts concerning the suitability of targeting p53 in the immunotherapy of cancer patients...
Clinical-scale lentiviral vector transduction of PBL for TCR gene therapy and potential for expression in less-differentiated cellsShicheng Yang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
J Immunother 31:830-9. 2008..Compared with gamma-retroviral vectors, the TCR transgene could be preferentially expressed on a less-differentiated cell population...
Lentiviral vector design for optimal T cell receptor gene expression in the transduction of peripheral blood lymphocytes and tumor-infiltrating lymphocytesStephanie Jones
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 20:630-40. 2009..This paper emphasizes the need to optimize both promoter function and protein synthesis in constructs that require stoichiometric production of multiple protein subunits...
Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1Paul F Robbins
National Institutes of Health, National Cancer Institute, Surgery Branch, Bethesda, MD 20892 1201, USA
J Clin Oncol 29:917-24. 2011....
A simple and effective method to generate lentiviral vectors for ex vivo gene delivery to mature human peripheral blood lymphocytesShicheng Yang
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther Methods 23:73-83. 2012..We describe a simple, efficient, and low-cost method for the production of clinical-grade LVV for ex vivo gene therapy protocols...
Primary human T lymphocytes engineered with a codon-optimized IL-15 gene resist cytokine withdrawal-induced apoptosis and persist long-term in the absence of exogenous cytokineCary Hsu
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 175:7226-34. 2005..In the setting of adoptive cell transfer, IL-15-transduced lymphocytes may prolong lymphocyte survival in vivo and could potentially enhance antitumor activity...
Genetic engineering of murine CD8+ and CD4+ T cells for preclinical adoptive immunotherapy studiesSid P Kerkar
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunother 34:343-52. 2011..These results indicate that preclinical murine models of adoptive immunotherapies are more practical using γ-retroviral rather than lentiviral vectors...
Primary human lymphocytes transduced with NY-ESO-1 antigen-specific TCR genes recognize and kill diverse human tumor cell linesYangbing Zhao
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 174:4415-23. 2005..These results strongly support the idea that redirection of normal T cell specificity by TCR gene transfer can have potential applications in tumor adoptive immunotherapy...
B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cellsJames N Kochenderfer
Experimental Transplantation and Immunology Branch, National Cancer Institute NCI, Bethesda, MD 20892, USA
Blood 119:2709-20. 2012....
IL-12 triggers a programmatic change in dysfunctional myeloid-derived cells within mouse tumorsSid P Kerkar
Center for Cancer Research, National Cancer Institute, NIH, Bethesda, Maryland 20892 1502, USA
J Clin Invest 121:4746-57. 2011....
High-efficiency transfection of primary human and mouse T lymphocytes using RNA electroporationYangbing Zhao
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Mol Ther 13:151-9. 2006..The results of this study indicate that mRNA electroporation provides a powerful tool to introduce genes into both human and murine primary T lymphocytes...
Eradication of B-lineage cells and regression of lymphoma in a patient treated with autologous T cells genetically engineered to recognize CD19James N Kochenderfer
Surgery Branch, National Cancer Institute, Bethesda, MD 20892, USA
Blood 116:4099-102. 2010..Adoptive transfer of anti-CD19-CAR-expressing T cells is a promising new approach for treating B-cell malignancies. This study is registered at www.clinicaltrials.gov as #NCT00924326...
Identification and characterization of a tumor infiltrating CD56(+)/CD16 (-) NK cell subset with specificity for pancreatic and prostate cancer cell linesTimothy L Frankel
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Cancer Immunol Immunother 59:1757-69. 2010..This represents the first report of CD56(+)CD16(-) NK cells with apparent specificity for pancreatic and prostate cancer cell lines and associated with tumor regression following the treatment with an immune modulating agent...
Lack of specific gamma-retroviral vector long terminal repeat promoter silencing in patients receiving genetically engineered lymphocytes and activation upon lymphocyte restimulationWilliam R Burns
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Blood 114:2888-99. 2009..The use of immunomodulatory adjuvants, eg, vaccination or cytokine therapy, for in vivo T-cell activation may help overcome this metabolic quiescence and thus augment cellular immunotherapy-based cancer therapy...
Improving adoptive T cell therapy by targeting and controlling IL-12 expression to the tumor environmentLing Zhang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Mol Ther 19:751-9. 2011..Notably, this targeted and controlled IL-12 treatment was without toxicity. Taken together, our results suggest that using the NFAT.hIL12.PA2 vector might be a promising approach to enhance adoptive cancer immunotherapy...
The position of the AUG start codon in MFG-based γ-retroviral vectors has a dramatic effect on translation-dependent protein expressionTimothy L Frankel
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
J Gene Med 13:478-86. 2011..Little is known about the impact of variations in start codon location within MFG-based vectors on protein expression...
T cells targeting carcinoembryonic antigen can mediate regression of metastatic colorectal cancer but induce severe transient colitisMaria R Parkhurst
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Mol Ther 19:620-6. 2011..It also emphasizes the destructive power of small numbers of highly avid T cells and the limitations of using CEA as a target for cancer immunotherapy...
A TCR targeting the HLA-A*0201-restricted epitope of MAGE-A3 recognizes multiple epitopes of the MAGE-A antigen superfamily in several types of cancerNachimuthu Chinnasamy
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 186:685-96. 2011..On the basis of these results, a clinical trial is planned in which patients bearing a variety of tumor histologies will receive autologous PBLs that have been transduced with this optimized anti-MAGE-A3 TCR...
Rapid production of clinical-grade gammaretroviral vectors in expanded surface roller bottles using a "modified" step-filtration process for clearance of packaging cellsSteven A Feldman
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 22:107-15. 2011..To date, this platform has generated five clinical-grade gammaretroviral vector products, four of which are now being used in adoptive cell therapy clinical trials for the treatment of a variety of solid cancers...
Transduction of an HLA-DP4-restricted NY-ESO-1-specific TCR into primary human CD4+ lymphocytesYangbing Zhao
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
J Immunother (1997) 29:398-406. 2006..Major histocompatibility complex class II TCR-transduced CD4 T cells provides an alternative source of tumor antigen-specific T cells for adoptive immunotherapy of cancer patients...
Cytokine-independent growth and clonal expansion of a primary human CD8+ T-cell clone following retroviral transduction with the IL-15 geneCary Hsu
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Blood 109:5168-77. 2007..It does not appear that the retroviral vector integration sites played a role in the continuous growth of this cell clone, but this remains under investigation...
Enhanced antitumor activity of T cells engineered to express T-cell receptors with a second disulfide bondCyrille J Cohen
Surgery Branch, Center for Cancer Research, National Cancer Institute NIH, 10 Center Drive, Bethesda, MD 20892, USA
Cancer Res 67:3898-903. 2007..Preferential pairing of cysteine-modified receptor chains accounts for these observations, which could have significant implications for the improvement of TCR gene therapy...
Enhanced antitumor activity of murine-human hybrid T-cell receptor (TCR) in human lymphocytes is associated with improved pairing and TCR/CD3 stabilityCyrille J Cohen
Surgery Branch, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, MD 20892-1201, USA
Cancer Res 66:8878-86. 2006..These findings have implications for human TCR gene transfer therapy and may provide new insights into the biology of the TCR/CD3 complex...
Development of human anti-murine T-cell receptor antibodies in both responding and nonresponding patients enrolled in TCR gene therapy trialsJeremy L Davis
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Clin Cancer Res 16:5852-61. 2010..We conducted 2 clinical trials in which cancer patients were treated with lymphocytes genetically engineered to express murine T-cell receptors (mTCR) specific for tumor-associated antigens p53 and gp100...
Real-time quantitative reverse transcriptase-polymerase chain reaction as a method for determining lentiviral vector titers and measuring transgene expressionGregory Lizee
Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 14:497-507. 2003....
Local delivery of interleukin-12 using T cells targeting VEGF receptor-2 eradicates multiple vascularized tumors in miceDhanalakshmi Chinnasamy
National Cancer Institute, Clinical Research Center, Bethesda, MD, USA
Clin Cancer Res 18:1672-83. 2012..We investigated the feasibility of delivering the proinflammatory cytokine interleukin (IL)-12 into tumor using T cells genetically engineered to express a chimeric antigen receptor (CAR) against the VEGF receptor-2 (VEGFR-2)...
Evaluation of γ-retroviral vectors that mediate the inducible expression of IL-12 for clinical applicationLing Zhang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
J Immunother 35:430-9. 2012..These results support the clinical application of adoptive transfer of young TIL engineered with the NFAT.hIL12 vector as a new approach for cancer immunotherapy...
Inhibition of histone lysine methylation enhances cancer-testis antigen expression in lung cancer cells: implications for adoptive immunotherapy of cancerMahadev Rao
Section of Thoracic Oncology, Surgery Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland 20892, USA
Cancer Res 71:4192-204. 2011..Combining DNA-demethylating agents with compounds, such as DZNep, that modulate histone lysine methylation may provide a novel epigenetic strategy to augment cancer-testis gene expression as an adjunct to adoptive cancer immunotherapy...
Preferential survival of CD4+ T lymphocytes engineered with anti-human immunodeficiency virus (HIV) genes in HIV-infected individualsRichard A Morgan
National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
Hum Gene Ther 16:1065-74. 2005..Thus, these data strongly support the hypothesis that anti-HIV genes afford a survival advantage to T cells and potential benefit to HIV-1+ individuals...
High-affinity TCRs generated by phage display provide CD4+ T cells with the ability to recognize and kill tumor cell linesYangbing Zhao
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 179:5845-54. 2007..Although the biological activity of these two relatively low-affinity TCRs was comparable to wild-type reactivity in CD8(+) T cells, introduction of these TCR dramatically increased the reactivity of CD4(+) T cells to tumor cell lines...
Modulating the differentiation status of ex vivo-cultured anti-tumor T cells using cytokine cocktailsShicheng Yang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Building 10, CRC 3 W 3864, Bethesda, MD, 20892, USA
Cancer Immunol Immunother 62:727-36. 2013..The methodology that we developed for generating a less-differentiated anti-tumor CD8+ T cells ex vivo may be ideal for the adoptive immunotherapy of cancer...
Adoptive cell transfer: a clinical path to effective cancer immunotherapySteven A Rosenberg
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, Maryland 20892, USA
Nat Rev Cancer 8:299-308. 2008....
The shedding of CD62L (L-selectin) regulates the acquisition of lytic activity in human tumor reactive T lymphocytesShicheng Yang
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, United States of America
PLoS ONE 6:e22560. 2011..The linkage of the shedding of CD62L from the surface of anti-tumor T cells and acquisition of lytic activity, suggests a new function for CD62L in T cell effector functions and anti-tumor activity...
Enhanced inhibition of human immunodeficiency virus type 1 replication by novel lentiviral vectors expressing human immunodeficiency virus type 1 envelope antisense RNAMario R Mautino
Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 10C103, Bethesda, MD 20892 1851, USA
Hum Gene Ther 13:1027-37. 2002..Our results suggest that higher vector copy number and increased levels of envAS RNA expression contribute to block replication of divergent strains of HIV-1...
Protein L: a novel reagent for the detection of chimeric antigen receptor (CAR) expression by flow cytometryZhili Zheng
Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Transl Med 10:29. 2012..Despite the improvements in the design of CARs and expansion of the number of target antigens, there is no universal flow cytometric method available to detect the expression of CARs on the surface of transduced lymphocytes...
Genetic engineering with T cell receptorsLing Zhang
National Institutes of Health, National Cancer Institute, Surgery Branch, Bethesda, MD 20892, USA
Adv Drug Deliv Rev 64:756-62. 2012..In this review, we discuss the progress and prospects of TCR gene-engineered T cells as a therapeutic strategy for treating patients with melanoma and other cancers...
Treating cancer with genetically engineered T cellsTristen S Park
National Institutes of Health, National Cancer Institute, Surgery Branch, Bethesda, MD 20892, USA
Trends Biotechnol 29:550-7. 2011..In this review, we discuss the development of TCR and CAR gene transfer technology and the expansion of these therapies into different cancers with the recent demonstration of the clinical efficacy of these treatments...
Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interferenceNatasha J Caplen
Medical Genetics Branch, National Human Genome Research Institute and Neurogenetics Branch, National Institute of Neurological Diseases, National Institutes of Health, Bethesda, MD 20892, USA
Hum Mol Genet 11:175-84. 2002..This study demonstrates the feasibility of targeting a transcript associated with an important group of genetic diseases by RNAi...
Toxicity of a first-generation adenoviral vector in rhesus macaquesJay N Lozier
National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA
Hum Gene Ther 13:113-24. 2002..The rhesus macaque may be a useful animal model in which to evaluate mechanisms of adenovirus toxicities that have been encountered during clinical gene therapy trials...
Inhibition of viral gene expression and replication in mosquito cells by dsRNA-triggered RNA interferenceNatasha J Caplen
National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, 20892, USA
Mol Ther 6:243-51. 2002....
Anti-CD22-chimeric antigen receptors targeting B-cell precursor acute lymphoblastic leukemiaWaleed Haso
Pediatric Oncology Branch, Center for Cancer Research CCR, National Cancer Institute NCI, National Institutes of Health NIH, Bethesda, MD 20892, USA
Blood 121:1165-74. 2013..We conclude that second-generation m971 mAb-derived anti-CD22 CARs are promising novel therapeutics that should be tested in BCP-ALL...
Cancer therapy with genetically-modified T cells for the treatment of melanomaKiran H Lagisetty
National Institutes of Health, National Cancer Institute, Surgery Branch, Bethesda, MD, USA
J Gene Med 14:400-4. 2012..This article is a US Government work and is in the public domain in the USA...
Gene therapy-based treatment for HIV-positive patients with malignanciesElizabeth M Kang
Molecular and Clinical Hematology Branch, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD 20892, USA
J Hematother Stem Cell Res 11:809-16. 2002..Vector-transduced cells remain detectable at low levels more than 3 years post-transplantation, suggesting the potential for gene therapy as a reasonable goal for the treatment of HIV...
High-efficiency lentiviral vector-mediated gene transfer into murine macrophages and activated splenic B lymphocytesGabriela R Rossi
National Cancer Institute, Building 10, Room 2B04, National Institutes of Health, 10 Center Drive, MSC 1502, Bethesda, MD 20892, USA
Hum Gene Ther 14:385-91. 2003..We conclude that HIV-1-based lentiviral vectors can mediate efficient gene transfer into primary murine macrophages and mature B lymphocytes...
Nonmyeloablative conditioning followed by transplantation of genetically modified HLA-matched peripheral blood progenitor cells for hematologic malignancies in patients with acquired immunodeficiency syndromeElizabeth M Kang
Molecular and Clinical Hematology Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA
Blood 99:698-701. 2002..These results suggest that nonmyeloablative allogeneic transplantation in the context of highly active antiretroviral therapy is feasible in patients with treatment-sensitive HIV infection...
Gene therapy of HIV-1 infection using lentiviral vectors expressing anti-HIV-1 genesMario R Mautino
Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA
AIDS Patient Care STDS 16:11-26. 2002..This review focuses on the use of lentiviral vectors as the main agents to mediate inhibition of HIV-1 replication and discusses the different genetic intervention strategies for gene therapy of HIV-1 infection...
Semliki Forest virus vectors for gene transferJarmo Wahlfors
A.I. Virtanen Institute for Molecular Sciences, University of Kuopio, Kuopio, Finland
Methods Mol Med 76:493-502. 2003
Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cellsMorvarid Moayeri
Department of Anatomy and Cell Biology, Flow Cytometry Core Facility, The George Washington University Medical Center, Washington, DC 20037, USA
Mol Ther 10:892-902. 2004..Notably, the hemophilic phenotype of all treated mice was corrected, thus demonstrating the potential of HSC-directed oncoretroviral-mediated factor VIII gene transfer as a curative therapeutic strategy for hemophilia A...
Minigene-containing retroviral vectors using an alphavirus/retrovirus hybrid vector system. Production and useJarmo Wahlfors
A.I. Virtanen Institute for Molecular Sciences, University of Kuopio, Kuopio, Finland
Methods Mol Med 69:173-86. 2002
Selecting highly affine and well-expressed TCRs for gene therapy of melanomaAnnelies Jorritsma
Department of Immunology, The Netherlands Cancer Institute NKI, Amsterdam, The Netherlands
Blood 110:3564-72. 2007..The procedures described in this study should be of general value for the selection of well- and stably expressed, high-affinity, and safe human TCRs for subsequent clinical testing...
