Potential of AAV vectors in the treatment of metabolic disease (Q37134001)

18 August 2017

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title

Potential of AAV vectors in the treatment of metabolic disease (English)

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18 August 2017

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I E Alexander

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18 August 2017

S C Cunningham

series ordinal

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18 August 2017

G J Logan

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18 August 2017

J Christodoulou

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18 August 2017

language of work or name

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10 April 2008

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18 August 2017

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18 August 2017

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18 August 2017

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18 August 2017

page(s)

831-839

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Clinical approach to treatable inborn metabolic diseases: an introduction

18 August 2017

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

21 January 2018

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

21 January 2018

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Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.

21 January 2018

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Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain

21 January 2018

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Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity

21 January 2018

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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

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Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

21 January 2018

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Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype

21 January 2018

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Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector

21 January 2018

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Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

21 January 2018

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Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors

21 January 2018

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Immune responses to adeno-associated virus vectors

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2008.64

21 January 2018

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CD8(+) T-cell responses to adeno-associated virus capsid in humans

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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https://api.crossref.org/works/10.1038%2FGT.2008.64

Immune responses to AAV in clinical trials

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2008.64

Intrahepatic immunity: a tale of two sites?

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Efficient induction of immune tolerance to coagulation factor IX following direct intramuscular gene transfer

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2008.64

Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions

21 January 2018

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Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2008.64

AAV vector integration sites in mouse hepatocellular carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

No evidence for tumorigenesis of AAV vectors in a large-scale study in mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Prevention of neuropathology in the mouse model of Hurler syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Adeno-associated virus-mediated transfer of human acid maltase gene results in a transient reduction of glycogen accumulation in muscle of Japanese quail with acid maltase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

PKU is a reversible neurodegenerative process within the nigrostriatum that begins as early as 4 weeks of age in Pah(enu2) mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria

21 January 2018

1 reference

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In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Urea-cycle disorders as a paradigm for inborn errors of hepatocyte metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Problems in the management of urea cycle disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Mutations and polymorphisms in the human ornithine transcarbamylase gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Liver failure and neurologic disease in a patient with homocystinuria

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Current role of liver transplantation for methylmalonic acidemia: a review of the literature

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Efficacy of living donor liver transplantation for patients with methylmalonic acidemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

The pyruvate dehydrogenase complex as a target for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Optic neuropathy induced by reductions in mitochondrial superoxide dismutase

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Adeno-associated virus-mediated gene transfer of the heart/muscle adenine nucleotide translocator (ANT) in mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Use of mitochondrial antioxidant defenses for rescue of cells with a Leber hereditary optic neuropathy-causing mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene transfer of wild-type apoA-I and apoA-I Milano reduce atherosclerosis to a similar extent

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene therapy for type I glycogen storage diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

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%2FGT.2008.64

Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Long-term rescue of a lethal inherited disease by adeno-associated virus-mediated gene transfer in a mouse model of molybdenum-cofactor deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene therapy for lipoprotein lipase deficiency: working toward clinical application

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

Gene delivery to the juvenile mouse liver using AAV2/8 vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Tolerance induction by viral in vivo gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Four new mutations in the ornithine transcarbamylase gene

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.64

7 January 2021

Identifiers

DOI

10.1038/GT.2008.64

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Dimensions Publication ID

18 August 2017

1026896374

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Fatcat ID

release_lagkfqgnwzaoxml5nzhyc623va

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Fatcat

https://api.fatcat.wiki/v0/release/lagkfqgnwzaoxml5nzhyc623va

24 November 2022

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