Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

Blood - Tập 122 Số 8 - Trang 1341-1349 - 2013
Hiroki Torikai1, Andreas Reik2, Frank Soldner3, Edus H. Warren4, Carrie Yuen1, Yuanyue Zhou2, Denise L. Crossland1,5, Helen Huls1, Nicholas Littman2, Ziying Zhang2, Scott S. Tykodi4, Partow Kebriaei6, Dean A. Lee1,5, Jeffrey C. Miller2, Edward J. Rebar2, Michael C. Holmes2, Rudolf Jaenisch7,3, Richard E. Champlin6, Philip D. Gregory2, Laurence J.N. Cooper1,5
1Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX
2Sangamo BioSciences Inc., Richmond, CA;
3The Whitehead Institute, Cambridge, MA;
4Program in Immunology, Fred Hutchinson Cancer Research Center, Seattle, WA;
5The University of Texas Graduate School of Biomedical Sciences and
6Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX; and
7Department of Biology, Massachusetts Institute of Technology, Cambridge, MA.

Tóm tắt

Key PointsAllogeneic-donor–derived cells can be genetically modified to eliminate expression of HLA-A. HLA-A disruption from donor cells is a step toward generating allogeneic cells as an off-the-shelf therapeutic.

Từ khóa


Tài liệu tham khảo

Gaspar, 2011, Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction., Sci Transl Med, 3, 97ra80, 10.1126/scitranslmed.3002716

Kalos, 2011, T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia., Sci Transl Med, 3, 95ra73, 10.1126/scitranslmed.3002842

Porter, 2011, Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia., N Engl J Med, 365, 725, 10.1056/NEJMoa1103849

Hacein-Bey-Abina, 2010, Efficacy of gene therapy for X-linked severe combined immunodeficiency., N Engl J Med, 363, 355, 10.1056/NEJMoa1000164

Rama, 2010, Limbal stem-cell therapy and long-term corneal regeneration., N Engl J Med, 363, 147, 10.1056/NEJMoa0905955

Petersdorf, 2001, Major-histocompatibility-complex class I alleles and antigens in hematopoietic-cell transplantation., N Engl J Med, 345, 1794, 10.1056/NEJMoa011826

Opelz, 1999, HLA compatibility and organ transplant survival. Collaborative Transplant Study., Rev Immunogenet, 1, 334

Früh, 1997, Inhibition of MHC class I antigen presentation by viral proteins., J Mol Med (Berl), 75, 18, 10.1007/s001090050082

Berger, 2000, Expression of herpes simplex virus ICP47 and human cytomegalovirus US11 prevents recognition of transgene products by CD8(+) cytotoxic T lymphocytes., J Virol, 74, 4465, 10.1128/JVI.74.10.4465-4473.2000

Riolobos

Figueiredo, 2006, Class-, gene-, and group-specific HLA silencing by lentiviral shRNA delivery., J Mol Med (Berl), 84, 425, 10.1007/s00109-005-0024-2

Gonzalez, 2005, Amplification of RNAi—targeting HLA mRNAs., Mol Ther, 11, 811, 10.1016/j.ymthe.2004.12.023

Haga, 2006, Permanent, lowered HLA class I expression using lentivirus vectors with shRNA constructs: Averting cytotoxicity by alloreactive T lymphocytes., Transplant Proc, 38, 3184, 10.1016/j.transproceed.2006.10.158

Sykulev, 1996, Evidence that a single peptide-MHC complex on a target cell can elicit a cytolytic T cell response., Immunity, 4, 565, 10.1016/S1074-7613(00)80483-5

Urnov, 2010, Genome editing with engineered zinc finger nucleases., Nat Rev Genet, 11, 636, 10.1038/nrg2842

Perez, 2008, Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases., Nat Biotechnol, 26, 808, 10.1038/nbt1410

O’Connor, 2012, Adoptive T-cell therapy improves treatment of canine non-Hodgkin lymphoma post chemotherapy., Sci Rep, 2, 249, 10.1038/srep00249

Manuri, 2010, piggyBac transposon/transposase system to generate CD19-specific T cells for the treatment of B-lineage malignancies., Hum Gene Ther, 21, 427, 10.1089/hum.2009.114

Singh, 2008, Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system., Cancer Res, 68, 2961, 10.1158/0008-5472.CAN-07-5600

Torikai, 2012, A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR., Blood, 119, 5697, 10.1182/blood-2012-01-405365

Doyon, 2010, Transient cold shock enhances zinc-finger nuclease-mediated gene disruption., Nat Methods, 7, 459, 10.1038/nmeth.1456

Guschin, 2010, A rapid and general assay for monitoring endogenous gene modification., Methods Mol Biol, 649, 247, 10.1007/978-1-60761-753-2_15

Lengner, 2010, Derivation of pre-X inactivation human embryonic stem cells under physiological oxygen concentrations., Cell, 141, 872, 10.1016/j.cell.2010.04.010

Soldner, 2009, Parkinson’s disease patient-derived induced pluripotent stem cells free of viral reprogramming factors., Cell, 136, 964, 10.1016/j.cell.2009.02.013

Hockemeyer, 2008, A drug-inducible system for direct reprogramming of human somatic cells to pluripotency., Cell Stem Cell, 3, 346, 10.1016/j.stem.2008.08.014

Johnson, 2003, Locus-specific constitutive and cytokine-induced HLA class I gene expression., J Immunol, 170, 1894, 10.4049/jimmunol.170.4.1894

Mori, 1997, HLA gene and haplotype frequencies in the North American population: the National Marrow Donor Program Donor Registry., Transplantation, 64, 1017, 10.1097/00007890-199710150-00014

Miller, 2007, An improved zinc-finger nuclease architecture for highly specific genome editing., Nat Biotechnol, 25, 778, 10.1038/nbt1319

Jena, 2010, Redirecting T-cell specificity by introducing a tumor-specific chimeric antigen receptor., Blood, 116, 1035, 10.1182/blood-2010-01-043737

Kebriaei, 2012, Infusing CD19-directed T cells to augment disease control in patients undergoing autologous hematopoietic stem-cell transplantation for advanced B-lymphoid malignancies., Hum Gene Ther, 23, 444, 10.1089/hum.2011.167

Kochenderfer, 2012, B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells., Blood, 119, 2709, 10.1182/blood-2011-10-384388

Brentjens, 2011, Safety and persistence of adoptively transferred autologous CD19-targeted T cells in patients with relapsed or chemotherapy refractory B-cell leukemias., Blood, 118, 4817, 10.1182/blood-2011-04-348540

Kochenderfer, 2010, Eradication of B-lineage cells and regression of lymphoma in a patient treated with autologous T cells genetically engineered to recognize CD19., Blood, 116, 4099, 10.1182/blood-2010-04-281931

Provasi, 2012, Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer., Nat Med, 18, 807, 10.1038/nm.2700

Singh, 2011, Reprogramming CD19-specific T cells with IL-21 signaling can improve adoptive immunotherapy of B-lineage malignancies., Cancer Res, 71, 3516, 10.1158/0008-5472.CAN-10-3843

Drukker, 2002, Characterization of the expression of MHC proteins in human embryonic stem cells., Proc Natl Acad Sci USA, 99, 9864, 10.1073/pnas.142298299

Brower, 1994, Minimal requirements for peptide mediated activation of CD8+ CTL., Mol Immunol, 31, 1285, 10.1016/0161-5890(94)90079-5

Felix, 2007, Specificity of T-cell alloreactivity., Nat Rev Immunol, 7, 942, 10.1038/nri2200

Cooper, 2012, Good T cells for bad B cells., Blood, 119, 2700, 10.1182/blood-2011-12-398719

Liu, 2010, Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases., Biotechnol Bioeng, 106, 97, 10.1002/bit.22654

Zhao, 2011, Immunogenicity of induced pluripotent stem cells., Nature, 474, 212, 10.1038/nature10135

Katagiri, 2011, Frequent loss of HLA alleles associated with copy number-neutral 6pLOH in acquired aplastic anemia., Blood, 118, 6601, 10.1182/blood-2011-07-365189

Parham, 2005, MHC class I molecules and KIRs in human history, health and survival., Nat Rev Immunol, 5, 201, 10.1038/nri1570

Borrego, 1998, Recognition of human histocompatibility leukocyte antigen (HLA)-E complexed with HLA class I signal sequence-derived peptides by CD94/NKG2 confers protection from natural killer cell-mediated lysis., J Exp Med, 187, 813, 10.1084/jem.187.5.813

Braud, 1998, HLA-E binds to natural killer cell receptors CD94/NKG2A, B and C., Nature, 391, 795, 10.1038/35869

Riteau, 2001, HLA-G1 co-expression boosts the HLA class I-mediated NK lysis inhibition., Int Immunol, 13, 193, 10.1093/intimm/13.2.193

Rouas-Freiss, 1997, The alpha1 domain of HLA-G1 and HLA-G2 inhibits cytotoxicity induced by natural killer cells: is HLA-G the public ligand for natural killer cell inhibitory receptors?, Proc Natl Acad Sci USA, 94, 5249, 10.1073/pnas.94.10.5249

Brickner, 2006, The PANE1 gene encodes a novel human minor histocompatibility antigen that is selectively expressed in B-lymphoid cells and B-CLL., Blood, 107, 3779, 10.1182/blood-2005-08-3501

Tykodi, 2008, C19orf48 encodes a minor histocompatibility antigen recognized by CD8+ cytotoxic T cells from renal cell carcinoma patients., Clin Cancer Res, 14, 5260, 10.1158/1078-0432.CCR-08-0028