Delivery technologies for T cell gene editing: Applications in cancer immunotherapy

Gene therapy trials for sickle cell disease halted after two patients develop cancer

(2021) Jocelyn Kaiser   SCIENCE

Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

(2020) Margaret M. Billingsley et al.   NANO LETTERS

Knocking out barriers to engineered cell activity

(2020) Jennifer R. Hamilton et al.   SCIENCE

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Gene editing and CRISPR in the clinic: current and future perspectives

(2020) Matthew P. Hirakawa et al.   BIOSCIENCE REPORTS

Signaling from T cell receptors (TCRs) and chimeric antigen receptors (CARs) on T cells

(2020) Ling Wu et al.   Cellular & Molecular Immunology

Targeting CD33 in chemoresistant AML patient-derived xenografts by CAR-CIK cells engineered with an optimized Sleeping Beauty transposon version

(2020) Maria Caterina Rotiroti et al.   MOLECULAR THERAPY

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Multi-phenotype CRISPR-Cas9 Screen Identifies p38 Kinase as a Target for Adoptive Immunotherapies

(2020) Devikala Gurusamy et al.   CANCER CELL

CRISPR-Mediated Non-Viral Site-Specific Gene Integration and Expression in T Cells: Protocol and Application for T-Cell Therapy

(2020) Zelda Odé et al.   Cancers

High-throughput continuous-flow microfluidic electroporation of mRNA into primary human T cells for applications in cellular therapy manufacturing

(2020) Charles A. Lissandrello et al.   Scientific Reports

Diversifying the structure of zinc finger nucleases for high-precision genome editing

(2019) David E. Paschon et al.   Nature Communications

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Granulocyte-macrophage colony-stimulating factor inactivation in CAR T-Cells prevents monocyte-dependent release of key cytokine release syndrome mediators

(2019) Mohit Sachdeva et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Disruption of CTLA-4 expression on peripheral blood CD8 + T cell enhances anti-tumor efficacy in bladder cancer

(2019) Wei Zhang et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

Regional delivery of mesothelin-targeted CAR T cells for pleural cancers: Safety and preliminary efficacy in combination with anti-PD-1 agent.

(2019) Prasad S. Adusumilli et al.   JOURNAL OF CLINICAL ONCOLOGY

Preclinical Evaluation of Allogeneic CAR T Cells Targeting BCMA for the Treatment of Multiple Myeloma

(2019) Cesar Sommer et al.   MOLECULAR THERAPY

Therapeutic potential of CRISPR/Cas9 gene editing in engineered T‐cell therapy

(2019) Qianqian Gao et al.   Cancer Medicine

CRISPR-Cas9 disruption of PD-1 enhances activity of universal EGFRvIII CAR T cells in a preclinical model of human glioblastoma

(2019) Bryan D. Choi et al.   Journal for ImmunoTherapy of Cancer

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

(2019) Pedro P.G. Guimaraes et al.   JOURNAL OF CONTROLLED RELEASE

Targeting REGNASE-1 programs long-lived effector T cells for cancer therapy

(2019) Jun Wei et al.   NATURE

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency

(2019) David N. Nguyen et al.   NATURE BIOTECHNOLOGY

Optimized DNA electroporation for primary human T cell engineering

(2018) Zhang Zhang et al.   BMC BIOTECHNOLOGY

CRISPR/Cas9-mediated knockout of DGK improves anti-tumor activities of human T cells

(2018) In-Young Jung et al.   CANCER RESEARCH

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Efficient Non-Viral T-Cell Engineering by Sleeping Beauty Minicircles Diminishing DNA Toxicity and miRNAs Silencing the Endogenous T-Cell Receptors

(2018) Julian Clauss et al.   HUMAN GENE THERAPY

Cationic polymers for non-viral gene delivery to human T cells

(2018) Brynn R. Olden et al.   JOURNAL OF CONTROLLED RELEASE

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

(2018) Matthew L Cooper et al.   LEUKEMIA

Long Terminal Repeat CRISPR-CAR-Coupled “Universal” T Cells Mediate Potent Anti-leukemic Effects

(2018) Christos Georgiadis et al.   MOLECULAR THERAPY

Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA

(2018) Rebecca L. Ball et al.   NANO LETTERS

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

(2018) Li Tang et al.   NATURE BIOTECHNOLOGY

Enhanced mRNA delivery into lymphocytes enabled by lipid-varied libraries of charge-altering releasable transporters

(2018) Colin J. McKinlay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CAR T cell immunotherapy for human cancer

(2018) Carl H. June et al.   SCIENCE

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

(2018) Joseph A. Fraietta et al.   NATURE

Induction of resistance to chimeric antigen receptor T cell therapy by transduction of a single leukemic B cell

(2018) Marco Ruella et al.   NATURE MEDICINE

CRISPR/Cas9-mediated PD-1 disruption enhances human mesothelin-targeted CAR T cell effector functions

(2018) Wanghong Hu et al.   CANCER IMMUNOLOGY IMMUNOTHERAPY

Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function

(2018) Eric Shifrut et al.   CELL

Non-viral therapeutic cell engineering with the Sleeping Beauty transposon system

(2018) Michael Hudecek et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

Cell engineering with microfluidic squeezing preserves functionality of primary immune cells in vivo

(2018) Tia DiTommaso et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CRISPR knock out CTLA-4 enhances the anti-tumor activity of cytotoxic T lymphocytes

(2017) Long Shi et al.   GENE

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 and Application of Zinc Finger Proteins and TALEs for Biomedical Research

(2017) Moon-Soo Kim et al.   MOLECULES AND CELLS

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

(2017) Justin Eyquem et al.   NATURE

In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers

(2017) Tyrel T. Smith et al.   Nature Nanotechnology

Delivery technologies for genome editing

(2017) Hao Yin et al.   NATURE REVIEWS DRUG DISCOVERY

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

(2017) Waseem Qasim et al.   Science Translational Medicine

Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers

(2017) H. F. Moffett et al.   Nature Communications

T cell-targeting nanoparticles focus delivery of immunotherapy to improve antitumor immunity

(2017) Daniela Schmid et al.   Nature Communications

A versatile system for rapid multiplex genome-edited CAR T cell generation

(2017) Jiangtao Ren et al.   Oncotarget

Genome-Editing Technologies in Adoptive T Cell Immunotherapy for Cancer

(2017) Nathan Singh et al.   Current Hematologic Malignancy Reports

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

CRISPR-Cas9 mediated LAG-3 disruption in CAR-T cells

(2017) Yongping Zhang et al.   Frontiers of Medicine

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Driving gene-engineered T cell immunotherapy of cancer

(2016) Laura A Johnson et al.   CELL RESEARCH

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

(2016) Jiangtao Ren et al.   CLINICAL CANCER RESEARCH

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

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

Enhanced CAR T-cell engineering using non-viral Sleeping Beauty transposition from minicircle vectors

(2016) R Monjezi et al.   LEUKEMIA

Stable, Nonviral Expression of Mutated Tumor Neoantigen-specific T-cell Receptors Using the Sleeping Beauty Transposon/Transposase System

(2016) Drew C Deniger et al.   MOLECULAR THERAPY

Redirecting Specificity of T cells Using the Sleeping Beauty System to Express Chimeric Antigen Receptors by Mix-and-Matching of VL and VH Domains Targeting CD123+ Tumors

(2016) Radhika Thokala et al.   PLoS One

Tethered IL-15 augments antitumor activity and promotes a stem-cell memory subset in tumor-specific T cells

(2016) Lenka V. Hurton et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Adoptive T Cell Therapies: A Comparison of T Cell Receptors and Chimeric Antigen Receptors

(2016) Daniel T. Harris et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Immunotherapy of acute leukemia by chimeric antigen receptor-modified lymphocytes using an improved Sleeping Beauty transposon platform

(2016) Chiara F. Magnani et al.   Oncotarget

CRISPR-Cas9-mediated disruption of PD-1 on human T cells for adoptive cellular therapies of EBV positive gastric cancer

(2016) Shu Su et al.   OncoImmunology

Clinical manufacturing of CAR T cells: foundation of a promising therapy

(2016) Xiuyan Wang et al.   Molecular Therapy-Oncolytics

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

(2015) Laurent Poirot et al.   CANCER RESEARCH

Genetic Engineering of T Cells to Target HERV-K, an Ancient Retrovirus on Melanoma

(2015) J. Krishnamurthy et al.   CLINICAL CANCER RESEARCH

Designing chimeric antigen receptors to effectively and safely target tumors

(2015) Michael C Jensen et al.   CURRENT OPINION IN IMMUNOLOGY

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

Error-Prone Repair of DNA Double-Strand Breaks

(2015) Kasey Rodgers et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma

(2015) Joal D Beane et al.   MOLECULAR THERAPY

Sleeping Beauty Transposition of Chimeric Antigen Receptors Targeting Receptor Tyrosine Kinase-Like Orphan Receptor-1 (ROR1) into Diverse Memory T-Cell Populations

(2015) Drew C. Deniger et al.   PLoS One

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

A Pilot Trial Using Lymphocytes Genetically Engineered with an NY-ESO-1-Reactive T-cell Receptor: Long-term Follow-up and Correlates with Response

(2014) P. F. Robbins et al.   CLINICAL CANCER RESEARCH

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

Nature of Tumor Control by Permanently and Transiently Modified GD2 Chimeric Antigen Receptor T Cells in Xenograft Models of Neuroblastoma

(2014) N. Singh et al.   Cancer Immunology Research

Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection

(2013) C. A. Didigu et al.   BLOOD

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

(2013) H. Torikai et al.   BLOOD

Sleeping Beauty System to Redirect T-cell Specificity for Human Applications

(2013) Sourindra N. Maiti et al.   JOURNAL OF IMMUNOTHERAPY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Comparison of Lentiviral and Sleeping Beauty Mediated αβ T Cell Receptor Gene Transfer

(2013) Anne-Christine Field et al.   PLoS One

A vector-free microfluidic platform for intracellular delivery

(2013) A. Sharei et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Tolerability of intramuscular and intradermal delivery by CELLECTRA®adaptive constant current electroporation device in healthy volunteers

(2013) Malissa C Diehl et al.   Human Vaccines & Immunotherapeutics

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

RNA-guided genetic silencing systems in bacteria and archaea

(2012) Blake Wiedenheft et al.   NATURE

FLASH assembly of TALENs for high-throughput genome editing

(2012) Deepak Reyon et al.   NATURE BIOTECHNOLOGY

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

(2012) Elena Provasi et al.   NATURE MEDICINE

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

Transposon-mediated BAC transgenesis in human ES cells

(2012) Maria Rostovskaya et al.   NUCLEIC ACIDS RESEARCH

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Generation and Genetic Engineering of Human Induced Pluripotent Stem Cells Using Designed Zinc Finger Nucleases

(2012) Sivaprakash Ramalingam et al.   STEM CELLS AND DEVELOPMENT

Infusing CD19-Directed T Cells to Augment Disease Control in Patients Undergoing Autologous Hematopoietic Stem-Cell Transplantation for Advanced B-Lymphoid Malignancies

(2011) Partow Kebriaei et al.   HUMAN GENE THERAPY

Engineering HIV-Resistant Human CD4+ T Cells with CXCR4-Specific Zinc-Finger Nucleases

(2011) Craig B. Wilen et al.   PLoS Pathogens

Nonviral Gene Delivery: Principle, Limitations, and Recent Progress

(2009) Mohammed S. Al-Dosari et al.   AAPS Journal

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

Construction and Preclinical Evaluation of an Anti-CD19 Chimeric Antigen Receptor

(2009) James N. Kochenderfer et al.   JOURNAL OF IMMUNOTHERAPY

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

Efficient gene targeting in Drosophila by direct embryo injection with zinc-finger nucleases

(2008) K. J. Beumer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA