Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

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COT drives resistance to RAF inhibition through MAP kinase pathway reactivation

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

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Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

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Bmi-1 collaborates with c-Myc in tumorigenesis by inhibiting c-Myc-induced apoptosis via INK4a/ARFPreprint

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Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

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Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

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Aberrant ERG expression cooperates with loss of PTEN to promote cancer progression in the prostate

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RNA polymerase II and the integration of nuclear eventsPreprint

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The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

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Rearrangements of the RAF kinase pathway in prostate cancer, gastric cancer and melanoma

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Oncogenic AKAP9-BRAF fusion is a novel mechanism of MAPK pathway activation in thyroid cancer

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New genes involved in cancer identified by retroviral tagging

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Cancer gene discovery in solid tumours using transposon-based somatic mutagenesis in the mouse

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Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

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RNA polymerase II is an essential mRNA polyadenylation factor

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Site-specific integration and tailoring of cassette design for sustainable gene transfer

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High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)

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RTCGD: retroviral tagged cancer gene database

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Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

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Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

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Genome-wide retroviral insertional tagging of genes involved in cancer in Cdkn2a-deficient mice

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A conditional transposon-based insertional mutagenesis screen for hepatocellular carcinoma-associated genes in mice

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In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance

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Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts

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Tumor progression locus 2 (Tpl-2) encodes a protein kinase involved in the progression of rodent T-cell lymphomas and in T-cell activation.

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Increased gene dosage of Ink4a/Arf results in cancer resistance and normal agingPreprint

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Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

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Structure and transforming potential of the human cot oncogene encoding a putative protein kinase.

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Aberrant Rheb-mediated mTORC1 activation and Pten haploinsufficiency are cooperative oncogenic eventsPreprint

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Tpl-2 acts in concert with Ras and Raf-1 to activate mitogen-activated protein kinase.

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The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors

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Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the cHS4 Insulator to a Lentiviral Vector

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The PTEN and INK4A/ARF tumor suppressors maintain myelolymphoid homeostasis and cooperate to constrain histiocytic sarcoma development in humans

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Combinatorial Incorporation of Enhancer-Blocking Components of the Chicken β-Globin 5′HS4 and Human T-Cell Receptor α/δ BEAD-1 Insulators in Self-Inactivating Retroviral Vectors Reduces Their Genotoxic Potential

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Genomic and Functional Assays Demonstrate Reduced Gammaretroviral Vector Genotoxicity Associated With Use of the cHS4 Chromatin Insulator

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Risk assessment in skin gene therapy: viral–cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors

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Mutational activation of the MAP3K8 protooncogene in lung cancer

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TPL2/COT/MAP3K8 (TPL2) Activation Promotes Androgen Depletion-Independent (ADI) Prostate Cancer Growth

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Structure and Transforming Potential of the Human cot Oncogene Encoding a Putative Protein Kinase

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Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region.

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Genetic dangers in poly(A) signals

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Viral vector platforms within the gene therapy landscape

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Clinical use of lentiviral vectors

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Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

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Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

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Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

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Viral vectors for therapy of neurologic diseases

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Viral Vectors: The Road to Reducing Genotoxicity

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Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β ^A(T87Q) - <i>Globin</i> Gene

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Recognition and inhibition of SARS-CoV-2 by humoral innate immunity pattern recognition molecules

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Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

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Retroviral and Lentiviral Safety Analysis of Gene-Modified T Cell Products and Infused HIV and Oncology Patients

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Three-dimensional brain-like microenvironments facilitate the direct reprogramming of fibroblasts into therapeutic neurons

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Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives

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Choosing the Right Tool for Genetic Engineering: Clinical Lessons from Chimeric Antigen Receptor-T Cells

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HIV-1-mediated insertional activation of STAT5B and BACH2 trigger viral reservoir in T regulatory cells

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Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases

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Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

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A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells

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Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

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Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges

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PSMA-Specific CAR-Engineered T Cells Eradicate Disseminated Prostate Cancer in Preclinical Models

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

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Enhancement of the viability of T cells electroporated with DNA via osmotic dampening of the DNA-sensing cGAS–STING pathway

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Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

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Systematic improvements in lentiviral transduction of primary human natural killer cells undergoing ex vivo expansion

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Recent trends in the gene therapy of β-thalassemia

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Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease

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Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

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Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

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New treatment for osteoarthritis: Gene therapy

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Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy

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Improved calvarial bone repair by hASCs engineered with Cre/loxP-based baculovirus conferring prolonged BMP-2 and MiR-148b co-expression

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Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalomyopathy

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Gene delivery to rat and human Schwann cells and nerve segments: a comparison of AAV 1–9 and lentiviral vectors

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The potential of gene therapy for recessive dystrophic epidermolysis bullosa*

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Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B

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In vivo engineering of CAR-T cells: delivery strategies and clinical translation

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Towards a Safer, More Randomized Lentiviral Vector Integration Profile Exploring Artificial LEDGF Chimeras

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Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells

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The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy

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Gene therapies for high-grade gliomas: from the bench to the bedside.

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Replication-incompetent gammaretroviral and lentiviral vector-based insertional mutagenesis screens identify prostate cancer progression genes

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Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector

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RNA Structure: Past, Future, and Gene Therapy Applications

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Integration Site and Clonal Expansion in Human Chronic Retroviral Infection and Gene Therapy

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RNA Virus-Based Episomal Vector with a Fail-Safe Switch Facilitating Efficient Genetic Modification and Differentiation of iPSCs

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Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment

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Genetic Targeting of the Albumin Locus to Treat Hemophilia

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Lentiviral Vector-based Insertional Mutagenesis Identifies Genes Involved in the Resistance to Targeted Anticancer Therapies

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Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders

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Insulators to Improve the Safety of Retroviral Vectors for HIV Gene Therapy

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AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization

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Correcting inborn errors of immunity: From viral mediated gene addition to gene editing

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Development and Preclinical Evaluation of an Integrase Defective Lentiviral Vector Vaccine Expressing the HIVACAT T Cell Immunogen in Mice

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529. Lentiviral Vectors with a Reduced Splicing Interference Potential Have a Significantly Improved Safety Profile In Vivo

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Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

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3. Safety Assessment of SIN LVs Harboring Chromatin Insulators in the Sensitive Cdkn2a-/- In Vivo Genotoxicity Assay Show Enhancer-Blocking Activity of Specific Insulator Sequences

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