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MOLECULAR THERAPY

Journal

Identifier:

E112282

ISSN:

1525-0016

Overview

Overview

Outputs (102)

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A Human IPSC-Based Model of Globoid Cell Leukodystrophy Uncovers Early Neurodevelopmental Defects

Abstract

A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

Academic Article

A Tolerizing mRNA Vaccine against Autoimmunity?

Academic Article

A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector)

Academic Article

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

Academic Article

AAV-mediated gene therapy for focal epilepsy by expressing neuropeptide Y and Y2 receptor in rodent and non-human primate hippocampus

Academic Article

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

Academic Article

Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer

Academic Article

Autologous Cell & Gene Therapy for the Therapeutic Targeting of Immune Payloads to the Solid Tumor Microenvironment: Preliminary Results of the TEM-GBM Study

Abstract

Axons Mediate the Distribution of Arylsulfatase a Within the Mouse Hippocampus Upon Gene Delivery

Academic Article

CD4 CAR T Cells Drive Extensive CD8 CAR T Cell Expansion, Leading to Severe Cytokine Release Syndrome

Abstract

COVID-eVax, an electroporated DNA vaccine candidate encoding the SARS-CoV-2 RBD, elicits protective responses in animal models

Academic Article

Cell-Specific Transcriptional Regulatory Domains Attract Gamma Retroviral Integration in the Human Genome

Abstract

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

Academic Article

Charting a clear path: The ASGCT standardized pathways conference

Academic Article

Chimeric Lysosomal Enzymes with Improved Bioavailability to Advance Gene Therapy Strategies for Globoid Cell Leukodystrophy

Abstract

Chimeric enzymes enhance treatment potential for globoid cell leukodystrophy through hematopoietic stem cell gene therapy

Academic Article

Clinical and functional recovery from experimental autoimmune encephalomyelitis by intracisternal delivery of IL4 from a helper-dependent adenoviral vector. A pre-clinical model of gene therapy of multiple sclerosis

Abstract

Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

Academic Article

Comparative Study of Retroviral Insertions in ADA-SCID Patients Treated with PBL-GT and HSC-GT Unveils a Cell Specific Integration Profile

Conference Paper

Correction of β-Thalassemia Through the Generation of Genomic Rearrangements in the β-Globin Locus

Abstract

Cyclosporin A and Rapamycin Relieve Distinct Lentiviral Restriction Blocks in Hematopoietic Stem and Progenitor Cells

Academic Article

Developing Allele-Specific Gene Editing Technologies for Silencing or Precise Correction of GFAP Mutations Causing Alexander's Disease

Abstract

Direct Intra-Brain LV-Mediated Gene Delivery Leads to Widespread Transgene Distribution and High-Level Enzymatic Correction in the Whole CNS of Twitcher Mice

Abstract

Dissecting the Transcriptional and Epigenetic Landscape of hiPSC-Derived Neural Stem and Progenitor Cells: Implications for Cell Therapy Approaches

Abstract

Dual Transgenesis of T Cells with a CD44v6 CAR and a Suicide Gene for the Safe Eradication of Myeloid Leukemia and Myeloma

Abstract

Dual Transgenesis of T Cells with a CD44v6 CAR and a Suicide Gene for the Safe Eradication of Myeloid Leukemia and Myeloma

Academic Article

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis

Academic Article

Editing human lumphocyte specificity for safe and effective adoptive immunotherapy

Conference Paper

Editing human lymphocyte specificity for safe and effective adoptive immunotherapy of leukemia

Conference Paper

Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector

Academic Article

Enforced IL-10 Expression Confers Type 1 Regulatory T Cell (Tr1) Phenotype and Function to Human CD4(+) T Cells

Academic Article

Erratum: "Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models" (Molecular Therapy (2009) vol. 17 (1073-1082) 10.1038/mt.2009.31)

Academic Article

Erratum: "Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich Syndrome patients leads to functional correction" (Molecular Therapy (2004) vol. 10 (903-915) 10.1016/j.ymthe.2004.08.008)

Academic Article

Erratum: IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism (Molecular Therapy (2017) 25(10) (2254–2269), (S1525001617303143), (10.1016/j.ymthe.2017.06.029))

Academic Article

Erratum: Preclinical Safety and Efficacy of Human CD34+ Cells Transduced with Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (Molecular Therapy (2013) 21 (175-184) DOI:10.1038/mt.2012.23)

Academic Article

Evidence for long-term efficacy and safety of gene therapy for Wiskott Aldrich syndrome in preclinical models

Academic Article

Extracellular Vesicles Containing IL-4 Modulate Neuroinflammation in a Mouse Model of Multiple Sclerosis

Academic Article

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Academic Article

Gene therapy for beta thalassemia: preclinical studies on human cells

Conference Paper

Gene transfer of modified idiotype into dendritic cells for immunotherapy of non-Hodgkin lymphomas

Abstract

Gene-Based Immune Reprogramming Overcomes the Immunosuppressive Microenvironment of Liver Metastases and Enables Protective T Cell Responses

Abstract

Generation of potent and stable human CD4(+) T regulatory cells by activation-independent expression of FOXP3

Academic Article

Genome-Wide Definition of Regulatory Regions and Transcripts During the Transition from Pluripotent to Neural-Restricted Stem Cells

Abstract

Graft-versus leukemia effect of HLA-haploidentical central-memory T cells expanded with leukemic APCs and modified with a suicide gene

Academic Article

Harnessing CD39 for the Treatment of Colorectal Cancer and Liver Metastases by Engineered T Cells

Abstract

Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy

Academic Article

IFNalpha by In Vivo-Engineered Macrophages Abates Liver Metastases and Triggers Counter Regulatory Responses Limiting Efficacy

Abstract

IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism

Academic Article

IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential

Academic Article

Immune System Regulation of Transgene Expression in the Brain 2: Differential Increase of mRNAs Encoding for Interferon-Regulated, Chemokine, and T-Cell Genes during Either Innate Acute or Chronic Systemic Adaptive Immune Responses to First Generation Adenoviral Vectors

Abstract

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

Academic Article

Investigating the Role of Innate Immune Signaling in AAV-Mediated Gene Transfer in hiPSC-Derived CNS Models

Abstract

Investigating the Role of Vector DNA Sensing and Transgene Expression on Innate Immune Signaling and Cell Toxicity upon AAV-Mediated Gene Transfer in hiPSC-Derived CNS Models

Abstract

Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues

Academic Article

Lentiviral Vector-Based Insertional Mutagenesis Identifies New Clinically Relevant Cancer Genes Involved in the Pathogenesis of Hepatocellular Carcinoma

Abstract

Lentiviral Vector-Based Insertional Mutagenesis Identifies New Oncogenes and Molecular Networks That Control Hepatocyte Transformation and Metabolism

Abstract

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

Academic Article

Lentiviral and retroviral vectors for transcriptionally targeted expression of human beta-globin

Conference Paper

Lentiviral vector gene therapy protects XCGD mice from acute Staphylococcus aureus pneumonia and inflammatory response

Academic Article

Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction

Academic Article

Lentiviral-transduced HSCs lead to long-term correction of ß-thalassemia by in vivo selection of erythroid progenitors

Conference Paper

Long term correction of beta thalassemia by transplantation of transduced hematopoietic stem cells

Conference Paper

Long-Term Immunological Profile and T Cell Dynamics in Patients Treated With Allogeneic Transplantation and TK-Cells for Hematological Malignancies

Abstract

Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs

Academic Article

Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs.

Academic Article

Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of alpha-L-Iduronidase in Mice With Mucopolysaccharidosis Type I

Academic Article

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

Academic Article

Mouse Models To Assess the Risk of Vector Insertional Mutagenesis upon Systemic Delivery

Abstract

N-Glycosylation Inhibition Hinders Immunosuppressive Activity of Tumor Microenvironment Cells and Improves CAR T Cell Efficacy

Abstract

Naive-Derived Memory Stem T Cells: A Novel Promising Platform for Cancer Immune-Gene Therapy

Conference Paper

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Academic Article

Persistent and Position Independent Transgene Expression in Erythroid Cells Transduced

Conference Paper

Preclinical Assessment of a Gene Therapy Approach to beta-Thalassemia

Conference Paper

ROBUST AND EFFICIENT REGULATION OF TRANSGENE EXPRESSION IN VIVO BY IMPROVED TETRACYCLINE-DEPENDENT LENTIVIRAL VECTORS

Academic Article

Regulated and Multiple miRNA and siRNA Delivery into Primary Cells by a Lentiviral Platform

Academic Article

Retroviral integrations in gene therapy trials

Academic Article

Safety of retroviral gene marking with a truncated NGF receptor

Abstract

Selective engraftment of genetically modified hematopoietic stem cells by a truncated erythropoietin receptor

Conference Paper

Semliki Forest Virus vectors with reduced cytotoxicity and temperature-sensitivity: long-term enhancement of transgene expression

Academic Article

Short-Hairpin RNA Interference with CD44v6 Reveals a Complex Role in Chemoresistance and Leukemogenicity

Abstract

Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting

Academic Article

Suicide Gene Therapy by Central Memory Human T Lymphocytes

Academic Article

T Cell Receptor Gene Transfer into Early Differentiated Lymphocytes by Lentiviral Vectors for Safe and Effective Adoptive Immune Therapy of Leukemia

Conference Paper

T-N/SCM Pre-Selection Generates CAR T Cell Products Driving Superior Anti-Tumor Responses While Curtailing Severe CRS

Abstract

TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD

Abstract

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.

Academic Article

Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease

Academic Article

The influence of vector design on integration site selection by gamma-retroviral and lentiviral vectors

Conference Paper

The suicide gene therapy challenge: How to improve a successful gene therapy approach

Academic Article

Therapeutic Efficacy of Hematopoietic Stem Cell Gene Therapy for Hurler Type 1 Mucopolysaccharidosis

Abstract

Towards the correction of the genetic defect in corneal keratinocytes from patients with macular corneal dystrophy type II

Conference Paper

Tracking the Fate and Activity of Individual HSC and Memory Stem T Cell Clones in GT Patients through Insertional Tagging

Conference Paper

Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design

Academic Article

Transduction of human CD34(+)CD38(-) bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors

Academic Article

Transduction of human hematopoietic stem cells and T-lymphocytes with RD114-TR-pseudotyped lentiviral vectors

Conference Poster

Transfection of RNA encoding tumor antigens following maturation of dendritic cells leads to prolonged presentation of antigen and the generation of high-affinity tumor-reactive cytotoxic T lymphocytes

Academic Article

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

Academic Article

Up to 10.5 Years of Follow-Up in 17 Subjects Treated with Hematopoietic Stem and Progenitor Cell Lentiviral Gene Therapy for Wiskott-Aldrich Syndrome

Abstract

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

Academic Article

Viral and Non-Viral Delivery of the CRISPR-Cas9 System in Human Hematopoietic Stem and Progenitor Cells

Abstract

dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice

Academic Article

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