(Q37140649)

18 August 2017

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title

Clinical gene therapy using recombinant adeno-associated virus vectors (English)

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18 August 2017

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C Mueller

1

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PubMed ID

18418415

18 August 2017

T R Flotte

series ordinal

2

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18 August 2017

17 April 2008

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18 August 2017

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18 August 2017

15

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18 August 2017

11

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18 August 2017

858-863

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https://scigraph.springernature.com/pub.10.1038/gt.2008.68

18 August 2017

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cites work

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

In vivo model of adeno-associated virus vector persistence and rescue

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Repeated Aerosolized AAV-CFTR for Treatment of Cystic Fibrosis: A Randomized Placebo-Controlled Phase 2B Trial

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Immune responses to AAV in a phase I study for Canavan disease.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Gene therapy for Parkinson's disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Practice parameter: thymectomy for autoimmune myasthenia gravis (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Pathophysiology of Parkinson's disease: the MPTP primate model of the human disorder.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Nerve growth factor expressed in the medial septum following in vivo gene delivery using a recombinant adeno-associated viral vector protects cholinergic neurons from fimbria-fornix lesion-induced degeneration

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Proteasome inhibition enhances AAV-mediated transgene expression in human synoviocytes in vitro and in vivo.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.68

7 January 2021

Identifiers

DOI

10.1038/GT.2008.68

1 reference

PubMed ID

18418415

Dimensions Publication ID

18 August 2017

0 references

1 reference

PubMed ID

18418415