(Q36994170)

17 August 2017

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title

Progress and prospects: gene therapy clinical trials (part 2). (English)

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17 August 2017

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17

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9

A M Leen

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4

M K Brenner

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17 August 2017

A Aiuti

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A C Bachoud-Lévi

2

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A Blesch

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F Cattaneo

5

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E A Chiocca

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G Gao

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K A High

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N R Lemoine

10

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I A McNeish

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G Meneguzzi

12

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M Peschanski

13

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M G Roncarolo

14

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D S Strayer

15

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M H Tuszynski

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J M Wilson

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1 November 2007

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1555-1563

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3303033

17 August 2017

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21 January 2018

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RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

21 January 2018

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Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice

21 January 2018

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Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the striatum of Huntington's disease transgenic mice

21 January 2018

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Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study.

21 January 2018

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Amelioration of cholinergic neuron atrophy and spatial memory impairment in aged rats by nerve growth factor.

21 January 2018

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Nontropic actions of neurotrophins: subcortical nerve growth factor gene delivery reverses age-related degeneration of primate cortical cholinergic innervation

21 January 2018

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A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease

21 January 2018

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Gene transfer in humans using a conditionally replicating lentiviral vector

21 January 2018

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Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells

21 January 2018

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A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency

21 January 2018

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Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

21 January 2018

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Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

21 January 2018

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

21 January 2018

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

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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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

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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

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Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

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

21 January 2018

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

21 January 2018

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Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

21 January 2018

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Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

21 January 2018

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Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

21 January 2018

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Adoptive immunotherapy for cancer: building on success

21 January 2018

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Dendritic-cell-based therapeutic vaccination against cancer

21 January 2018

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Re-engineered CD40 receptor enables potent pharmacological activation of dendritic-cell cancer vaccines in vivo.

21 January 2018

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Cancer regression in patients after transfer of genetically engineered lymphocytes

21 January 2018

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Primary human T lymphocytes engineered with a codon-optimized IL-15 gene resist cytokine withdrawal-induced apoptosis and persist long-term in the absence of exogenous cytokine

21 January 2018

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A phase I trial of intravenous CG7870, a replication-selective, prostate-specific antigen-targeted oncolytic adenovirus, for the treatment of hormone-refractory, metastatic prostate cancer.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

A phase I study of OncoVEXGM-CSF, a second-generation oncolytic herpes simplex virus expressing granulocyte macrophage colony-stimulating factor.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

Intratumoral recombinant GM-CSF-encoding virus as gene therapy in patients with cutaneous melanoma

21 January 2018

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An optimized clinical regimen for the oncolytic virus PV701.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

Introduction to the background, principles, and state of the art in suicide gene therapy.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

Phase I study of MetXia-P450 gene therapy and oral cyclophosphamide for patients with advanced breast cancer or melanoma.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme.

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

Corticostriatopallidal neuroprotection by adenovirus-mediated ciliary neurotrophic factor gene transfer in a rat model of progressive striatal degeneration

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

7 January 2021

Nerve growth factor promotes survival of septal cholinergic neurons after fimbrial transections

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

7 January 2021

Long-term survival and concomitant gene expression of ribozyme-transduced CD4+ T-lymphocytes in HIV-infected patients

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

7 January 2021

Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

7 January 2021

Prostate cancer gene therapy clinical trials

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303033

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3303033

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Dimensions Publication ID

17 August 2017

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1 reference