(Q36957474)

17 August 2017

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title

Progress and prospects: gene therapy clinical trials (part 1). (English)

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17 August 2017

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17

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17 August 2017

Alexander BL

1

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17 August 2017

Ali RR

2

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17 August 2017

Alton EW

3

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17 August 2017

Bainbridge JW

4

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17 August 2017

Braun S

5

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17 August 2017

Cheng SH

6

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17 August 2017

Flotte TR

7

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17 August 2017

Gaspar HB

8

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17 August 2017

Grez M

9

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17 August 2017

Griesenbach U

10

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17 August 2017

Kaplitt MG

11

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17 August 2017

Ott MG

12

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17 August 2017

Seger R

13

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17 August 2017

Simons M

14

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17 August 2017

Thrasher AJ

15

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17 August 2017

Thrasher AZ

16

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17 August 2017

publication date

1 October 2007

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17 August 2017

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17 August 2017

volume

14

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17 August 2017

issue

20

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17 August 2017

page(s)

1439-1447

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Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir

17 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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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Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.

21 January 2018

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Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Gene therapy: the 'pro-sense' approach to Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation

21 January 2018

1 reference

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Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.

21 January 2018

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Multiple reduced-intensity conditioning regimens facilitate correction of Fabry mice after transplantation of transduced cells

21 January 2018

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Gene therapy for lysosomal storage diseases.

21 January 2018

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Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Gene therapy: X-SCID transgene leukaemogenicity

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Chronic granulomatous disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Direct intramyocardial plasmid vascular endothelial growth factor-A165 gene therapy in patients with stable severe angina pectoris A randomized double-blind placebo-controlled study: the Euroinject One trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Angiogenic gene therapy in patients with nonrevascularizable ischemic heart disease: a phase 2 randomized, controlled trial of AdVEGF(121) (AdVEGF121) versus maximum medical treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Angiogenesis: where do we stand now?

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Increased vascular endothelial growth factor expression but impaired vascular endothelial growth factor receptor signaling in the myocardium of type 2 diabetic patients with chronic coronary heart disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Increased vascularity detected by digital subtraction angiography after VEGF gene transfer to human lower limb artery: a randomized, placebo-controlled, double-blinded phase II study

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Regional angiogenesis with vascular endothelial growth factor in peripheral arterial disease: a phase II randomized, double-blind, controlled study of adenoviral delivery of vascular endothelial growth factor 121 in patients with disabling intermitt

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Treatment with intramuscular vascular endothelial growth factor gene compared with placebo for patients with diabetes mellitus and critical limb ischemia: a double-blind randomized trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

7 January 2021

Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

7 January 2021

Gene therapy for chronic granulomatous disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

7 January 2021

Results of PREVENT III: a multicenter, randomized trial of edifoligide for the prevention of vein graft failure in lower extremity bypass surgery

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3303001

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3303001

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Dimensions Publication ID

17 August 2017

1009596933

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1 reference