(Q36446948)

14 August 2017

title

Gene therapy for treatment of inherited haematological disorders (English)

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14 August 2017

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Roland W Herzog

1

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14 August 2017

Ou Cao

series ordinal

2

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14 August 2017

J Nathan Hagstrom

series ordinal

3

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14 August 2017

Lixin Wang

series ordinal

4

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14 August 2017

language of work or name

English

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publication date

1 May 2006

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Expert Opinion on Biological Therapy

14 August 2017

published in

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14 August 2017

volume

6

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14 August 2017

issue

5

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14 August 2017

page(s)

509-522

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Gene therapy clinical trials worldwide 1989-2004-an overview.

14 August 2017

cites work

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

21 January 2018

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

21 January 2018

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

21 January 2018

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The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID

21 January 2018

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Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans

21 January 2018

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The molecular basis of X-linked severe combined immunodeficiency: defective cytokine receptor signaling

21 January 2018

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Naturally occurring primary deficiencies of the immune system

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Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency.

21 January 2018

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Gene therapy for severe combined immunodeficiency

21 January 2018

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Carrier detection in X-linked severe combined immunodeficiency based on patterns of X chromosome inactivation

21 January 2018

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Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor.

21 January 2018

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

21 January 2018

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Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial

21 January 2018

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Gene therapy. As Gelsinger case ends, gene therapy suffers another blow

21 January 2018

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Gene therapy insertional mutagenesis insights

21 January 2018

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Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

21 January 2018

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Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis.

21 January 2018

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Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells

21 January 2018

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Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia

21 January 2018

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Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Efficacy of gene therapy for SCID is being confirmed

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Failure of SCID-X1 gene therapy in older patients

21 January 2018

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The importance of adenosine deaminase for lymphocyte development and function.

21 January 2018

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Adenosine deaminase deficiency with mosaicism for a "second-site suppressor" of a splicing mutation: decline in revertant T lymphocytes during enzyme replacement therapy

21 January 2018

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T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

21 January 2018

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Molecular analysis of T lymphocyte-directed gene therapy for adenosine deaminase deficiency: long-term expression in vivo of genes introduced with a retroviral vector

21 January 2018

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Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

21 January 2018

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

21 January 2018

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

The innate immune response to adenovirus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Adeno-associated viruses undergo substantial evolution in primates during natural infections

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Correction of murine hemophilia A by hematopoietic stem cell gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Clinical gene transfer studies for hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Factor IX variants improve gene therapy efficacy for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

AAV-mediated gene transfer for treatment of hemophilia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Identification of mouse AAV capsid-specific CD8+ T cell epitopes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Evidence that DNase I hypersensitive site 5 of the human beta-globin locus control region functions as a chromosomal insulator in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Correction of sickle cell disease in transgenic mouse models by gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Expression of an anti-sickling beta-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Marking and gene expression by a lentivirus vector in transplanted human and nonhuman primate CD34(+) cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Lentivirus vector-mediated hematopoietic stem cell gene transfer of common gamma-chain cytokine receptor in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Induction of transgene-specific immunological tolerance in myeloablated nonhuman primates using lentivirally transduced CD34+ progenitor cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Clinical relevance of MGMT in the treatment of cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Cytokines, including stem cell factor alone, enhance lentiviral transduction in nondividing human LTCIC and NOD/SCID repopulating cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC)

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

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/10.1517%2F14712598.6.5.509

Intrinsic defects of B cell function in X-linked severe combined immunodeficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Atypical X-Linked Severe Combined Immunodeficiency Due to Possible Spontaneous Reversion of the Genetic Defect in T Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Murine leukemia induced by retroviral gene marking

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.6.5.509

21 January 2018

Identifiers

DOI

10.1517/14712598.6.5.509

1 reference

14 August 2017

1 reference