Gene editing for immune cell therapies

Logic-Gated ROR1 Chimeric Antigen Receptor Expression Rescues T Cell-Mediated Toxicity to Normal Tissues and Enables Selective Tumor Targeting

(2019) Shivani Srivastava et al.   CANCER CELL

CRISPRed Macrophages for Cell-Based Cancer Immunotherapy

(2018) Moumita Ray et al.   BIOCONJUGATE CHEMISTRY

Universal Chimeric Antigen Receptors for Multiplexed and Logical Control of T Cell Responses

(2018) Jang Hwan Cho et al.   CELL

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia

(2018) Miriam Y. Kim et al.   CELL

Induction of Neoantigen-specific Cytotoxic T Cells and Construction of T-cell Receptor-engineered T cells for Ovarian Cancer

(2018) Tatsuo Matsuda et al.   CLINICAL CANCER RESEARCH

An “off-the-shelf” fratricide-resistant CAR-T for the treatment of T cell hematologic malignancies

(2018) Matthew L Cooper et al.   LEUKEMIA

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

Absence of Replication-Competent Lentivirus in the Clinic: Analysis of Infused T Cell Products

(2018) Kenneth Cornetta et al.   MOLECULAR THERAPY

Optimization of IL13Rα2-Targeted Chimeric Antigen Receptor T Cells for Improved Anti-tumor Efficacy against Glioblastoma

(2018) Christine E. Brown et al.   MOLECULAR THERAPY

Enhancing T cell therapy through TCR-signaling-responsive nanoparticle drug delivery

(2018) Li Tang et al.   NATURE BIOTECHNOLOGY

Immune recognition of somatic mutations leading to complete durable regression in metastatic breast cancer

(2018) Nikolaos Zacharakis et al.   NATURE MEDICINE

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

(2018) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antitumor Activity Associated with Prolonged Persistence of Adoptively Transferred NY-ESO-1 c259 T Cells in Synovial Sarcoma

(2018) Sandra P. D'Angelo et al.   Cancer Discovery

Generation of Tumor Antigen-Specific iPSC-Derived Thymic Emigrants Using a 3D Thymic Culture System

(2018) Raul Vizcardo et al.   Cell Reports

CAR-T Cells Surface-Engineered with Drug-Encapsulated Nanoparticles Can Ameliorate Intratumoral T-cell Hypofunction

(2018) Natnaree Siriwon et al.   Cancer Immunology Research

Chimeric Antigen Receptor Expressing Natural Killer Cells for the Immunotherapy of Cancer

(2018) Rohtesh S. Mehta et al.   Frontiers in Immunology

Reprogramming human T cell function and specificity with non-viral genome targeting

(2018) Theodore L. Roth et al.   NATURE

Disruption of TET2 promotes the therapeutic efficacy of CD19-targeted T cells

(2018) Joseph A. Fraietta et al.   NATURE

Targeted delivery of a PD-1-blocking scFv by CAR-T cells enhances anti-tumor efficacy in vivo

(2018) Sarwish Rafiq et al.   NATURE BIOTECHNOLOGY

Genetic mechanisms of target antigen loss in CAR19 therapy of acute lymphoblastic leukemia

(2018) Elena J. Orlando et al.   NATURE MEDICINE

Tumor Antigen Escape from CAR T-cell Therapy

(2018) Robbie G. Majzner et al.   Cancer Discovery

Long-term safety and activity of axicabtagene ciloleucel in refractory large B-cell lymphoma (ZUMA-1): a single-arm, multicentre, phase 1–2 trial

(2018) Frederick L Locke et al.   LANCET ONCOLOGY

Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma

(2018) Stephen J. Schuster et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetically Engineered Macrophages: A Potential Platform for Cancer Immunotherapy

(2017) Kara W. Moyes et al.   HUMAN GENE THERAPY

Preclinical targeting of aggressive T-cell malignancies using anti-CD5 chimeric antigen receptor

(2017) K H Chen et al.   LEUKEMIA

Cord blood NK cells engineered to express IL-15 and a CD19-targeted CAR show long-term persistence and potent antitumor activity

(2017) E Liu et al.   LEUKEMIA

Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells

(2017) Daniel T. MacLeod et al.   MOLECULAR THERAPY

Engineering HIV-Resistant, Anti-HIV Chimeric Antigen Receptor T Cells

(2017) Malika Hale et al.   MOLECULAR THERAPY

Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection

(2017) Justin Eyquem et al.   NATURE

HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells

(2017) Germán G Gornalusse et al.   NATURE BIOTECHNOLOGY

Trivalent CAR T cells overcome interpatient antigenic variability in glioblastoma

(2017) Kevin Bielamowicz et al.   NEURO-ONCOLOGY

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

(2017) Sattva S. Neelapu et al.   NEW ENGLAND JOURNAL OF MEDICINE

A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma

(2017) Donald M. O’Rourke et al.   Science Translational Medicine

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

(2017) Waseem Qasim et al.   Science Translational Medicine

Targeting T-cell malignancies using anti-CD4 CAR NK-92 cells

(2017) Kevin G. Pinz et al.   Oncotarget

CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells

(2017) Levi J. Rupp et al.   Scientific Reports

Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS

(2017) Anjie Zhen et al.   PLoS Pathogens

Fine and Predictable Tuning of TALEN Gene Editing Targeting for Improved T Cell Adoptive Immunotherapy

(2017) Anne-Sophie Gautron et al.   Molecular Therapy-Nucleic Acids

T cells expressing an anti-B-cell maturation antigen chimeric antigen receptor cause remissions of multiple myeloma

(2016) S. A. Ali et al.   BLOOD

FVIII-specific human chimeric antigen receptor T-regulatory cells suppress T- and B-cell responses to FVIII

(2016) Jeongheon Yoon et al.   BLOOD

PD-1 blockade modulates chimeric antigen receptor (CAR)–modified T cells: refueling the CAR

(2016) Elise A. Chong et al.   BLOOD

Acquisition of a CD19-negative myeloid phenotype allows immune escape of MLL-rearranged B-ALL from CD19 CAR-T-cell therapy

(2016) R. Gardner et al.   BLOOD

Prospects for chimeric antigen receptor (CAR) γδ T cells: A potential game changer for adoptive T cell cancer immunotherapy

(2016) Hamid Reza Mirzaei et al.   CANCER LETTERS

A Chimeric Switch-Receptor Targeting PD1 Augments the Efficacy of Second-Generation CAR T Cells in Advanced Solid Tumors

(2016) Xiaojun Liu et al.   CANCER RESEARCH

Loss of the HVEM Tumor Suppressor in Lymphoma and Restoration by Modified CAR-T Cells

(2016) Michael Boice et al.   CELL

Precision Tumor Recognition by T Cells With Combinatorial Antigen-Sensing Circuits

(2016) Kole T. Roybal et al.   CELL

Engineering T Cells with Customized Therapeutic Response Programs Using Synthetic Notch Receptors

(2016) Kole T. Roybal et al.   CELL

Multiplex Genome Editing to Generate Universal CAR T Cells Resistant to PD1 Inhibition

(2016) Jiangtao Ren et al.   CLINICAL CANCER RESEARCH

Putting a price tag on novel autologous cellular therapies

(2016) Mohamed Abou-El-Enein et al.   CYTOTHERAPY

CD19 CAR–T cells of defined CD4+:CD8+ composition in adult B cell ALL patients

(2016) Cameron J. Turtle et al.   JOURNAL OF CLINICAL INVESTIGATION

Phase I trials using Sleeping Beauty to generate CD19-specific CAR T cells

(2016) Partow Kebriaei et al.   JOURNAL OF CLINICAL INVESTIGATION

Tandem CAR T cells targeting HER2 and IL13Rα2 mitigate tumor antigen escape

(2016) Meenakshi Hegde et al.   JOURNAL OF CLINICAL INVESTIGATION

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

(2016) Mark J Osborn et al.   MOLECULAR THERAPY

Prospective identification of neoantigen-specific lymphocytes in the peripheral blood of melanoma patients

(2016) Alena Gros et al.   NATURE MEDICINE

T-Cell Transfer Therapy Targeting Mutant KRAS in Cancer

(2016) Eric Tran et al.   NEW ENGLAND JOURNAL OF MEDICINE

Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease

(2016) C. T. Ellebrecht et al.   SCIENCE

CD19 CAR immune pressure induces B-precursor acute lymphoblastic leukaemia lineage switch exposing inherent leukaemic plasticity

(2016) Elad Jacoby et al.   Nature Communications

Chimeric antigen receptor T cells secreting anti-PD-L1 antibodies more effectively regress renal cell carcinoma in a humanized mouse model

(2016) Eloah Rabello Suarez et al.   Oncotarget

Treatment of the Immune-Related Adverse Effects of Immune Checkpoint Inhibitors

(2016) Claire F. Friedman et al.   JAMA Oncology

Tuning Sensitivity of CAR to EGFR Density Limits Recognition of Normal Tissue While Maintaining Potent Antitumor Activity

(2015) H. G. Caruso et al.   CANCER RESEARCH

Eradication of Large Solid Tumors by Gene Therapy with a T-Cell Receptor Targeting a Single Cancer-Specific Point Mutation

(2015) M. Leisegang et al.   CLINICAL CANCER RESEARCH

“Velcro” Engineering of High Affinity CD47 Ectodomain as Signal Regulatory Protein α (SIRPα) Antagonists That Enhance Antibody-dependent Cellular Phagocytosis

(2015) Chia Chi M. Ho et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Selective Inhibition of Tumor Growth by Clonal NK Cells Expressing an ErbB2/HER2-Specific Chimeric Antigen Receptor

(2015) Kurt Schönfeld et al.   MOLECULAR THERAPY

NY-ESO-1–specific TCR–engineered T cells mediate sustained antigen-specific antitumor effects in myeloma

(2015) Aaron P Rapoport et al.   NATURE MEDICINE

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

(2015) Jianbin Wang et al.   NUCLEIC ACIDS RESEARCH

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

Convergence of Acquired Mutations and Alternative Splicing of CD19 Enables Resistance to CART-19 Immunotherapy

(2015) E. Sotillo et al.   Cancer Discovery

Dual targeting of glioblastoma with chimeric antigen receptor-engineered natural killer cells overcomes heterogeneity of target antigen expression and enhances antitumor activity and survival

(2015) Sabrina Genßler et al.   OncoImmunology

Designed ankyrin repeat proteins are effective targeting elements for chimeric antigen receptors

(2015) Joanne A. Hammill et al.   Journal for ImmunoTherapy of Cancer

Drug-Loaded Nanoparticle Systems And Adult Stem Cells: A Potential Marriage For The Treatment Of Malignant Glioma?

(2015) Brenda Auffinger et al.   Oncotarget

Neural Stem Cell-Mediated Intratumoral Delivery of Gold Nanorods Improves Photothermal Therapy

(2014) Rachael Mooney et al.   ACS Nano

Engineered antigen-specific human regulatory T cells: immunosuppression of FVIII-specific T- and B-cell responses

(2014) Y. C. Kim et al.   BLOOD

Stem Cells Loaded With Multimechanistic Oncolytic Herpes Simplex Virus Variants for Brain Tumor Therapy

(2014) Matthias Duebgen et al.   JNCI-Journal of the National Cancer Institute

Reversal of Tumor Immune Inhibition Using a Chimeric Cytokine Receptor

(2014) Ann M Leen et al.   MOLECULAR THERAPY

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia

(2014) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cancer Immunotherapy Based on Mutation-Specific CD4+ T Cells in a Patient with Epithelial Cancer

(2014) E. Tran et al.   SCIENCE

Targeting CD20+ Aggressive B-cell Non-Hodgkin Lymphoma by Anti-CD20 CAR mRNA-Modified Expanded Natural Killer Cells In Vitro and in NSG Mice

(2014) Y. Chu et al.   Cancer Immunology Research

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

(2013) H. Torikai et al.   BLOOD

T Lymphocytes Expressing a CD16 Signaling Receptor Exert Antibody-Dependent Cancer Cell Killing

(2013) K. Kudo et al.   CANCER RESEARCH

Regeneration of Human Tumor Antigen-Specific T Cells from iPSCs Derived from Mature CD8 + T Cells

(2013) Raul Vizcardo et al.   Cell Stem Cell

Generation of Rejuvenated Antigen-Specific T Cells by Reprogramming to Pluripotency and Redifferentiation

(2013) Toshinobu Nishimura et al.   Cell Stem Cell

Hematopoietic stem cells for cancer immunotherapy

(2013) Eric Gschweng et al.   IMMUNOLOGICAL REVIEWS

Complete regression of glioblastoma by mesenchymal stem cells mediated prodrug gene therapy simulating clinical therapeutic scenario

(2013) Cestmir Altaner et al.   INTERNATIONAL JOURNAL OF CANCER

Systemically delivered measles virus-infected mesenchymal stem cells can evade host immunity to inhibit liver cancer growth

(2013) Hooi-Tin Ong et al.   JOURNAL OF HEPATOLOGY

CS1-specific chimeric antigen receptor (CAR)-engineered natural killer cells enhance in vitro and in vivo antitumor activity against human multiple myeloma

(2013) J Chu et al.   LEUKEMIA

Transfection of chimeric anti-CD138 gene enhances natural killer cell activation and killing of multiple myeloma cells

(2013) Hua Jiang et al.   Molecular Oncology

Allelic Exclusion and Peripheral Reconstitution by TCR Transgenic T Cells Arising From Transduced Human Hematopoietic Stem/Progenitor Cells

(2013) Francesca Giannoni et al.   MOLECULAR THERAPY

Generation of tumor-targeted human T lymphocytes from induced pluripotent stem cells for cancer therapy

(2013) Maria Themeli et al.   NATURE BIOTECHNOLOGY

Neural Stem Cell-Mediated Enzyme/Prodrug Therapy for Glioma: Preclinical Studies

(2013) K. S. Aboody et al.   Science Translational Medicine

PD-1- and CTLA-4-Based Inhibitory Chimeric Antigen Receptors (iCARs) Divert Off-Target Immunotherapy Responses

(2013) V. D. Fedorov et al.   Science Translational Medicine

CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia

(2013) R. J. Brentjens et al.   Science Translational Medicine

Therapeutic Efficacy and Fate of Bimodal Engineered Stem Cells in Malignant Brain Tumors

(2013) Jordi Martinez-Quintanilla et al.   STEM CELLS

The Timing of Neural Stem Cell-Based Virotherapy Is Critical for Optimal Therapeutic Efficacy When Applied With Radiation and Chemotherapy for the Treatment of Glioblastoma

(2013) Alex L. Tobias et al.   Stem Cells Translational Medicine

Tumor-targeted T cells modified to secrete IL-12 eradicate systemic tumors without need for prior conditioning

(2012) H. J. Pegram et al.   BLOOD

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

(2012) H. Torikai et al.   BLOOD

A Universal Strategy for Adoptive Immunotherapy of Cancer through Use of a Novel T-cell Antigen Receptor

(2012) K. Urbanska et al.   CANCER RESEARCH

Targeting Tumor-Infiltrating Macrophages Decreases Tumor-Initiating Cells, Relieves Immunosuppression, and Improves Chemotherapeutic Responses

(2012) J. B. Mitchem et al.   CANCER RESEARCH

Replication-Competent Retroviruses in Gene-Modified T Cells Used in Clinical Trials: Is It Time to Revise the Testing Requirements?

(2012) Adham S Bear et al.   MOLECULAR THERAPY

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

(2012) Elena Provasi et al.   NATURE MEDICINE

Safety and Activity of Anti–PD-L1 Antibody in Patients with Advanced Cancer

(2012) Julie R. Brahmer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety, Activity, and Immune Correlates of Anti–PD-1 Antibody in Cancer

(2012) Suzanne L. Topalian et al.   NEW ENGLAND JOURNAL OF MEDICINE

Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T Cells

(2012) J. Scholler et al.   Science Translational Medicine

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

(2011) J. N. Kochenderfer et al.   BLOOD

Durable Complete Responses in Heavily Pretreated Patients with Metastatic Melanoma Using T-Cell Transfer Immunotherapy

(2011) S. A. Rosenberg et al.   CLINICAL CANCER RESEARCH

Generation of dendritic cells and macrophages from human induced pluripotent stem cells aiming at cell therapy

(2011) S Senju et al.   GENE THERAPY

Comparison of mRNA and lentiviral based transfection of natural killer cells with chimeric antigen receptors recognizing lymphoid antigens

(2011) Laurent Boissel et al.   LEUKEMIA & LYMPHOMA

Encapsulated therapeutic stem cells implanted in the tumor resection cavity induce cell death in gliomas

(2011) Timo M Kauer et al.   NATURE NEUROSCIENCE

Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia

(2011) David L. Porter et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mesenchymal stem cells as cellular vehicles for delivery of nanoparticles to brain tumors

(2010) Mathilde Roger et al.   BIOMATERIALS

T Cells Targeting Carcinoembryonic Antigen Can Mediate Regression of Metastatic Colorectal Cancer but Induce Severe Transient Colitis

(2010) Maria R Parkhurst et al.   MOLECULAR THERAPY

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

(2009) L. A. Johnson et al.   BLOOD

Reprogramming T Lymphocytes for Melanoma Adoptive Immunotherapy by T-Cell Receptor Gene Transfer with Lentiviral Vectors

(2009) S. Bobisse et al.   CANCER RESEARCH

Efficient nonviral Sleeping Beauty transposon-based TCR gene transfer to peripheral blood lymphocytes confers antigen-specific antitumor reactivity

(2009) P D Peng et al.   GENE THERAPY

Transfection with mRNA for CD19 specific chimeric antigen receptor restores NK cell mediated killing of CLL cells

(2009) Laurent Boissel et al.   LEUKEMIA RESEARCH

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Engineering antigen-specific primary human NK cells against HER-2 positive carcinomas

(2008) A. Kruschinski et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA