HUMAN GENE THERAPY

Rivista

Codice:

E078680

ISSN:

1043-0342

Dati Generali

Pubblicazioni (125)

A New-Generation Stable Inducible Packaging Cell Line for Lentiviral Vectors

Articolo

A clinical protocol for gene transfer into peripheral blood lymphocytes for in vivo immunomodulation of donor anti-tumor immunity in patients affected by recurrent disease after allogeneic bone marrow transplantation

Articolo

A clinical protocol for transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA deficient SCID

Articolo

A glimpse into the in vivo dynamics during immune reconstitution in ADA-SCID patients after gene therapy treatment

Abstract

A human immunodeficiency virus type 1 pol gene-derived sequence (cPPT/CTS) increases the efficiency of transduction of human nondividing monocytes and T lymphocytes by lentiviral vectors

Articolo

A new chimeric enzyme for fluorouridine activation in cancer suicide gene therapy

Abstract

A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors

Articolo

A retroviral vector containing a muscle-specific enhancer drives gene expression only in differentiated muscle fibers

Articolo

Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies

Articolo

An innovative platform approach for the development of ex-vivo gene therapies for the treatment of lysosomal storage diseases with skeletal involvement

Abstract

Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings

Articolo

Bone marrow as source of hematopoietic stem cells for human gene therapy of beta thalassemia

Articolo

CD44v6 is associated with chemoresistance induced by bone marrow-derived mesenchymal stromal cells

Abstract

Cancer Gene Therapy: Present and Future.

Articolo

Cell therapy with genetically modified lymphocytes in onco-hematology

Contributo in Atti di convegno

Cell-surface marking of CD(34+)-restricted phenotypes of human hematopoietic progenitor cells by retrovirus-mediated gene transfer

Articolo

Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells

Articolo

Central Nervous system delivery of Interleukin 4 by a nonreplicative herpes simplex type 1 viral vector ameliorates autoimmune demyelination

Articolo

Characterization of potential genomic "safe harbor'' for efficient targeted gene addition with zinc finger nucleases

Contributo in Atti di convegno

Clinical applications of gene therapy for primary immunodeficiencies

Articolo

Clinical impact of suicide gene therapy in allogeneic hematopoietic stem cell transplantation

Articolo

Clinical protocol: transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID

Articolo

Combining Site-Specific Integration and Cassette Design to Achieve Robust Expression Without Impacting Endogenous Gene Expression

Contributo in Atti di convegno

Combining Targeted Integration and Cassette Design for Robust and Benign Transgene Expression without Impacting Endogenous Gene Transcription

Contributo in Atti di convegno

Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor

Articolo

Complete enzymatic correction of CNS tissue following intracerebral LV-mediated gene therapy in a murine model of GLD

Abstract

Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of Ex Vivo Gene Therapy: Review of Nonproliferative and Proliferative Changes

Articolo

Cytokine-Induced Killer Cells Engineered with Exogenous T-Cell Receptors Directed Against Melanoma Antigens: Enhanced Efficacy of Effector Cells Endowed with a Double Mechanism of Tumor Recognition

Articolo

Delivery to the central nervous system of a nonreplicative herpes simplex type 1 vector engineered with the interleukin 4 gene protects rhesus monkeys from hyperacute autoimmune encephalomyelitis

Articolo

Development of editing technologies for allele-specific silencing or precise correction of mutation hotspots affecting Alexander disease's patients

Abstract

Direct antigen presentation by leukemic dendritic cells promotes expansion of allogeneic leukemia-reactive T lymphocytes

Abstract

Early sulfatide storage and progressive neuronal and glial cell dysfunction revealed by human iPSC-based models of metachromatic leukodystrophy are rescued by lentiviral-mediated restoration of ARSA activity

Abstract

Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia

Abstract

Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia

Abstract

Editing central memory T lymphocyte specificity for safe and effective adoptive immunotherapy of leukemia

Contributo in Atti di convegno

Editorial - Gene Therapy in Europe

Articolo

Effective retrovirus-mediated gene transfer in normal and mutant human melanocytes

Articolo

Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation

Articolo

Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent

Articolo

Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors

Articolo

Efficient lentiviral vector-mediated delivery of hARSA in the brain of juvenile non-human primates

Abstract

Epigenome-editing strategies to enhance oligodendroglial differentiation from human induced pluripotent stem cells

Abstract

Exploiting targeted epigenetic editing to increase the yield, homogeneity and purity of human iPSCderived oligodendrocyte cell populations

Abstract

Exploring the identity and safety of hiPSC-derived neural stem/progenitor cells: implications for cell therapy approaches

Abstract

Fighting rare diseases: the model of the telethon research institutes in Italy

Articolo

Gene Therapy of Storage Disorders by Retroviral and Lentiviral Vectors

Articolo

Gene editing of naive and central memory T lymphocyte specificities for adoptive immune therapy of leukemia

Poster

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector

Articolo

Gene therapy for leukemia: alloreactivity and beyond

Contributo in Atti di convegno

Gene therapy trial with lentiviral vector transduced CD34+cells for the treatment of Wiskott-Aldrich Syndrome

Abstract

Genetically Modified Donor Leukocyte Transfusion and Graft-Versus-Leukemia Effect After Allogeneic Stem Cell Transplantation

Articolo

Genome Editing in Engineered T Cells for Cancer Immunotherapy

Articolo

Genome-wide analyses of regulatory regions and transcripts in hiPSC-derived neural stem/progenitor cells to define their safety and efficacy in cell therapy approaches for neurological disorders

Abstract

Genome-wide definition of regulatory regions and transcripts during the transition from pluripotent to neural restricted stem cells

Abstract

Good news on the clinical gene transfer front

Recensione

Graft–versus-Leukemia effect after allogeneic stem cell transplantation and transduced donor leukocyte transfusion

Articolo

GvHD kinetics after haploidentical TK-cells: in-vivo HSV-TK suicide machinery is effective in GvHD control and provides a long-term immune-suppressive treatment-free survival

Abstract

HIV-1-Derived Lentiviral Vectors Directly Activate Plasmacytoid Dendritic Cells, Which in Turn Induce the Maturation of Myeloid Dendritic Cells

Articolo

HSC gene therapy trial for Metachromatic Leukodystrophy: first report on gene marking efficiency

Abstract

Harnessing CD1-restricted T cells for the immunotherapy of leukemia

Abstract

Herpes simplex virus thymidine kinase gene transfer for controlled graft-versus-host disease and graft-versus-leukemia: Clinical follow-up and improved new vectors

Articolo

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

Abstract

High-resolution profiling of MLV integration in human CD34 cells reveals cell-specific transcriptional regulatory element

Articolo

Improving gene therapy approaches for globoid cell leukodystrophy: pre-clinical testing of chimeric GALC enzymes with enhanced bioavailability in human neural and hematopoietic cells

Abstract

Improving gene therapy approaches for globoid cell leukodystrophy: pre-clinical testing of chimeric GALC enzymes with enhanced bioavailability in human neural and hematopoietic cells

Abstract

In vivo models to assess the risk of insertional mutagenesis in the liver upon vector systemic delivery

Abstract

In vivo mouse models for vector genotoxicity testing and lentiviral vector-based cancer gene discovery

Articolo

In vivo selection of hematopoietic stem cells by a truncated erythropoietin receptor (tEpoR)

Contributo in Atti di convegno

In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors

Articolo

Inflammation converts immunologically inert human mesoangioblasts into sensitizers and targets of the alloreactive response: implications for allogeneic cell therapy of DMD

Abstract

Infusions of HSV-TK engineered donor lymphocytes promote the expansion of host-restricted viral-specific T lymphocytes and protect patients undergoing haploidentical stem cell transplantation from infectious mortality

Abstract

Inhibition of tumor growth by intramuscular injection of cDNA encoding tumor necrosis factor alpha coupled to NGR and RGD tumor-homing peptides

Articolo

Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS model

Abstract

Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS models

Abstract

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical Ex Vivo Gene Therapy Application

Articolo

Lentiviral Transduction of Primary Myeloma Cells with CD80 and CD154 Generates Anti-Myeloma Effector T Cells

Articolo

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 liver cancer genes and molecular networks that have a pivotal role in human hepatocarcinogenesis

Abstract

Lentivirus-induced dendritic cells for immunization against high-risk WT1(+) acute myeloid leukemia.

Articolo

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

Articolo

Long term persistence of TK-cells and dynamics of memory T cells in patients treated with suicide gene therapy for hematological malignancies

Abstract

Long term persistence of TK-cells and dynamics of memory T cells in patients treated with suicide gene therapy for hematological malignancies

Abstract

Long-term safety and correction of immune and metabolic defects in ADA-SCID children treated with gene therapy

Articolo

Memory T cells masquerading as naive cells: Implications on adoptive T cell immunotherapy

Poster

Mobilized blood CD341 cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice

Articolo

Modelling globoid cell leukodystrophy using integration-free human induced pluripotent stem cells (hiPSC)

Abstract

Neuronal and glial cells derived from a murine model of GM2-gangliosidosis display typical hallmarks of pathology and represent a valuable model to test gene therapy strategies

Abstract

Novel combined gene/cell therapy strategies to provide full rescue of the Sandhoff pathological phenotype

Abstract

Optimized bicistronic lentiviral vectors to correct b-hexosaminidase deficiency in neural and hematopoietic stem/progenitor cells and progeny

Abstract

Patient-specific gene-corrected iPSC-derived neural stem/progenitor cells for autologous cell therapy applications in lysosomal storage diseases

Abstract

Patient-specific induced pluripotent stem cells (iPSCs) to model metachromatic leukodystrophy

Abstract

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

Articolo

Realizing the Potential of Gene Therapies for Rare and Ultra-Rare Inherited Diseases

Articolo

Report on the 2nd annual meeting of the European Working Group on Human Gene Transfer and Therapy, London, November 18-21, 1994

Articolo

Restoration of bacterial killing activity of human respiratory cystic fibrosis cells through cationic vector-mediated cystic fibrosis transmembrane conductance regulator gene transfer

Articolo

Retroviral vector-mediated gene transfer into human primary myogenic cells leads to expression into muscle fibers in vivo

Articolo

Reversal of diabetes in mice by implantation of human fibroblasts genetically engineered to release mature human insulin.

Articolo

Reversible Immortalization of Human Myogenic Cells by Site-Specific Excision of a Retrovirally Transferred Oncogene

Articolo

Safety of arylsulfatase A over-expression for gene therapy of metachromatic leukodystrophy

Articolo

Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic anti-vector responses

Articolo

Suicide gene therapy of GvHD fully preserves immunocompetence after haploidentical hemopoietic cell transplantation

Abstract

Suicide gene therapy with central memory human T lymphocytes for a graft-versus-leukemia effect and control of graft-versus-host disease

Abstract

Targeting Integration to Selected Genomic Loci and In Situ Tailoring of Cassette Design Allows Robust Transgene Expression without Perturbing Endogenous Transcription

Contributo in Atti di convegno

Targeting chemoresistant myeloid leukaemia and multiple myeloma through genetic redirection of T cells against CD44 variant 6. Presented at the 19th Annual Meeting of the European Society of Gene and Cellular Therapy

Contributo in Atti di convegno

Targeting chemoresistant myeloid leukemia and multiple myeloma through genetic redirection of T cells against CD44 variant 6

Contributo in Atti di convegno

Targeting of myelogenous leukemia stem cells through genetic redirection of T cells against CD44v6

Abstract

Targeting of myeloid leukaemia stem cells through genetic redirection of T-cells against CD44v6

Abstract

The Renaissance of Gene and Cell Therapy: Florence 2016

Articolo

The Role of Conditioning in Hematopoietic Stem-Cell Gene Therapy

Articolo

The genotoxic potential of lentiviral vector integration is modulated by the interplay between vector design and mouse genotype

Articolo

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection

Abstract

The importance of be(ginn)ing Naive: implications for cancer immune-gene therapy

Contributo in Atti di convegno

Therapeutic advantage of combined gene/cell therapy strategies in the murine model of Sandhoff disease

Abstract

Therapeutic potential of genetically modified neural stem cells (NSCs) in a mouse model of Globoid Cell Leukodystrophy (GLD)

Abstract

Thymic renewal in adults after haploidentical hematopoietic stem cell transplantation and suicide gene therapy

Abstract

Toward epidermal stem cell-mediated ex vivo gene therapy of junctional epidermiolysis bullosa

Articolo

Tracking genetically engineered lymphocytes long-term reveals the dynamic of T-cell immunological memory

Abstract

Tracking hematopoietic stem cell fate in humans by retroviral tagging

Contributo in Atti di convegno

Tracking individual hematopoietic stem cell activity in vivo in humans through integration site barcoding

Abstract

Transduced fibroblasts and Metachromatic Leukodystrophy Lymphocytes Transfer Arylsulfatase A to Myelinating Glia and Deficient Cells In Vitro

Articolo

Transfer of the HSV-tk gene into donor peripheral blood lymphocytes for in vivo modulation of donor anti-tumor immunity after allogeneic bone marrow transplantation

Articolo

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug

Articolo

Uncovering haematopoietic system dynamics in gene therapy treated patients by retroviral tagging

Abstract

Various cells retrovirally transduced with N-acetylgalactosamine-6-sulphate sulphatase correct morquio skin fibroblasts in vitro

Articolo

Vector design influences retroviral integration in human hematopoietic cells

Contributo in Atti di convegno

No Results Found

HUMAN GENE THERAPY

Dati Generali

Pubblicazioni (125)