Metabolic engineering generates a transgene-free safety switch for cell therapy

Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation

(2020) Volker Wiebking et al.   HAEMATOLOGICA

Hypoimmunogenic derivatives of induced pluripotent stem cells evade immune rejection in fully immunocompetent allogeneic recipients

(2019) Tobias Deuse et al.   NATURE BIOTECHNOLOGY

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

(2019) Renata M. Martin et al.   Cell Stem Cell

Introduction of Exogenous HSV-TK Suicide Gene Increases Safety of Keratinocyte-Derived Induced Pluripotent Stem Cells by Providing Genetic “Emergency Exit” Switch

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Next-generation biocontainment systems for engineered organisms

(2018) Jeong Wook Lee et al.   Nature Chemical Biology

CRISPR/Cas9 genome editing in human hematopoietic stem cells

(2018) Rasmus O Bak et al.   Nature Protocols

The global landscape of cancer cell therapy

(2018) Jun Tang et al.   NATURE REVIEWS DRUG DISCOVERY

Selective targeting of engineered T cells using orthogonal IL-2 cytokine-receptor complexes

(2018) Jonathan T. Sockolosky et al.   SCIENCE

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Insights into the Mutational Burden of Human Induced Pluripotent Stem Cells from an Integrative Multi-Omics Approach

(2018) Matteo D’Antonio et al.   Cell Reports

Expanding the Therapeutic Window for CAR T Cell Therapy in Solid Tumors: The Knowns and Unknowns of CAR T Cell Biology

(2018) Keisuke Watanabe et al.   Frontiers in Immunology

Two-Dimensional Regulation of CAR-T Therapy with Orthogonal Switches

(2018) MyLinh T. Duong et al.   Molecular Therapy-Oncolytics

Engineering mammalian cells for disease diagnosis and treatment

(2018) Ana P Teixeira et al.   CURRENT OPINION IN BIOTECHNOLOGY

Drug-induced amino acid deprivation as strategy for cancer therapy

(2017) Marcus Kwong Lam Fung et al.   Journal of Hematology & Oncology

Human pluripotent stem cells recurrently acquire and expand dominant negative P53 mutations

(2017) Florian T. Merkle 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

Increasing the safety and efficacy of chimeric antigen receptor T cell therapy

(2017) Hua Li et al.   Protein & Cell

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

(2017) Rasmus O Bak et al.   eLife

Gene Therapy for “Bubble Boy” Disease

(2016) Jonathan Hoggatt   CELL

FDA Approval: Uridine Triacetate for the Treatment of Patients Following Fluorouracil or Capecitabine Overdose or Exhibiting Early-Onset Severe Toxicities Following Administration of These Drugs

(2016) G. Ison et al.   CLINICAL CANCER RESEARCH

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Toxicity and management in CAR T-cell therapy

(2016) Challice L Bonifant et al.   Molecular Therapy-Oncolytics

Gene therapy for primary immunodeficiencies

(2015) A. Fischer et al.   CLINICAL GENETICS

Self-Destruct Genetic Switch to Safeguard iPS Cells

(2015) Zoltán Ivics   MOLECULAR THERAPY

An Inducible Caspase-9 Suicide Gene to Improve the Safety of Therapy Using Human Induced Pluripotent Stem Cells

(2015) Shigeki Yagyu et al.   MOLECULAR THERAPY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Amino acid auxotrophy as a system of immunological control nodes

(2015) Peter J Murray   NATURE IMMUNOLOGY

An engineered bacterium auxotrophic for an unnatural amino acid: a novel biological containment system

(2015) Yusuke Kato   PeerJ

A Safeguard System for Induced Pluripotent Stem Cell-Derived Rejuvenated T Cell Therapy

(2015) Miki Ando et al.   Stem Cell Reports

The unfolded protein response governs integrity of the haematopoietic stem-cell pool during stress

(2014) Peter van Galen et al.   NATURE

CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences

(2014) Y. Lin et al.   NUCLEIC ACIDS RESEARCH

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

Development of an inducible caspase-9 safety switch for pluripotent stem cell–based therapies

(2014) Chuanfeng Wu et al.   Molecular Therapy-Methods & Clinical Development

Tumorigenicity as a clinical hurdle for pluripotent stem cell therapies

(2013) Andrew S Lee et al.   NATURE MEDICINE

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

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

(2012) Christine Aurnhammer et al.   Human Gene Therapy Methods

A transgene-encoded cell surface polypeptide for selection, in vivo tracking, and ablation of engineered cells

(2011) X. Wang et al.   BLOOD

A composite MyD88/CD40 switch synergistically activates mouse and human dendritic cells for enhanced antitumor efficacy

(2011) Priyadharshini Narayanan et al.   JOURNAL OF CLINICAL INVESTIGATION

Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine

(2011) Matthew Porteus   MOLECULAR THERAPY

Eliminating Cells Gone Astray

(2011) Michel Sadelain   NEW ENGLAND JOURNAL OF MEDICINE

Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy

(2011) Antonio Di Stasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enhanced Uridine Bioavailability Following Administration of a Triacetyluridine-Rich Nutritional Supplement

(2011) Melissa E. Weinberg et al.   PLoS One

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I–II study

(2009) Fabio Ciceri et al.   LANCET ONCOLOGY