Current progress on gene therapy for primary immunodeficiencies (Q38110363)

29 August 2017

title

Current progress on gene therapy for primary immunodeficiencies (English)

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29 August 2017

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L Zhang

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29 August 2017

A J Thrasher

series ordinal

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29 August 2017

H B Gaspar

series ordinal

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29 August 2017

publication date

30 May 2013

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29 August 2017

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29 August 2017

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29 August 2017

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29 August 2017

page(s)

963-969

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How I treat ADA deficiency

29 August 2017

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

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

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Failure of SCID-X1 gene therapy in older patients

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Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

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Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency

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Adenosine and the auditory system.

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Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

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Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

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Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

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Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

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Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency.

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Chronic granulomatous disease.

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Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease

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Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

21 January 2018

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Gene therapy of chronic granulomatous disease: the engraftment dilemma

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Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

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Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

21 January 2018

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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

21 January 2018

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Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy

21 January 2018

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In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

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Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics

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Transcription start regions in the human genome are favored targets for MLV integration

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Transcription factor binding sites are genetic determinants of retroviral integration in the human genome

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Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells

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

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Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

21 January 2018

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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

21 January 2018

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

21 January 2018

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Foamy-virus-mediated gene transfer to canine repopulating cells

21 January 2018

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Alpharetroviral self-inactivating vectors: long-term transgene expression in murine hematopoietic cells and low genotoxicity.

21 January 2018

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Self-inactivating alpharetroviral vectors with a split-packaging design

21 January 2018

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Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression

21 January 2018

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Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.

21 January 2018

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Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

21 January 2018

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Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

21 January 2018

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

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Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

21 January 2018

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Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells

21 January 2018

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

21 January 2018

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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.

21 January 2018

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Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene

21 January 2018

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Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.

21 January 2018

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Characterization of the Human Artemis Promoter by Heterologous Gene Expression In Vitro and In Vivo

21 January 2018

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Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.

21 January 2018

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Gene Therapy for Canine Leukocyte Adhesion Deficiency with Lentiviral Vectors Using the Murine Stem Cell Virus and Human Phosphoglycerate Kinase Promoters

21 January 2018

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Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter

21 January 2018

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In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals

21 January 2018

1 reference

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SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

21 January 2018

1 reference

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Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Correction of SCID-X1 using an enhancerless Vav promoter.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.

21 January 2018

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Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing

21 January 2018

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Optimal conditions for lentiviral transduction of engrafting human CD34+ cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Gene therapy for primary immunodeficiencies: Looking ahead, toward gene correction

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Enhancing Gene Targeting with Designed Zinc Finger Nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Correction of sickle cell disease by homologous recombination in embryonic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

In vivo genome editing using a high-efficiency TALEN system

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2013.21

FLASH assembly of TALENs for high-throughput genome editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Restoration of anti-Aspergillus defense by neutrophil extracellular traps in human chronic granulomatous disease after gene therapy is calprotectin-dependent

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Gene therapy for primary immunodeficiencies

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

Primary immunodeficiencies

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

7 January 2021

Gene therapy for chronic granulomatous disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

7 January 2021

A third-generation lentivirus vector with a conditional packaging system

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

7 January 2021

Development of a self-inactivating lentivirus vector.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2013.21

7 January 2021

Identifiers

DOI

10.1038/GT.2013.21

1 reference

29 August 2017

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