Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges (Q37866086)

24 August 2017

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title

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges (English)

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24 August 2017

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Federico Mingozzi

1

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PubMed ID

21499295

24 August 2017

Katherine A High

series ordinal

2

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24 August 2017

1 May 2011

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24 August 2017

Nature Reviews Genetics

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24 August 2017

12

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24 August 2017

341-355

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24 August 2017

5

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https://scigraph.springernature.com/pub.10.1038/nrg2988

24 August 2017

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Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes

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Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo

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Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets

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Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

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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

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

31 March 2020

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Dystrophin immunity in Duchenne's muscular dystrophy.

31 March 2020

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Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle.

31 March 2020

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

31 March 2020

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Investigation of chronic hepatitis C infection in individuals with haemophilia: assessment of invasive and non-invasive methods

31 March 2020

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

31 March 2020

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

31 March 2020

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Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients

31 March 2020

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

31 March 2020

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

31 March 2020

1 reference

Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector

31 March 2020

1 reference

Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector

31 March 2020

1 reference

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

31 March 2020

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Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

31 March 2020

1 reference

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

31 March 2020

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Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle

31 March 2020

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AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

31 March 2020

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The danger model: a renewed sense of self

31 March 2020

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Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

31 March 2020

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Manufacturing and characterizing AAV-based vectors for use in clinical studies

31 March 2020

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Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

31 March 2020

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Genome editing with engineered zinc finger nucleases

31 March 2020

1 reference

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

31 March 2020

1 reference

One of three successfully treated CGD patients in a Swiss-German gene therapy trial died due to his underlying disease: A position statement from the European Society of Gene Therapy (ESGT).

31 March 2020

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

31 March 2020

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

31 March 2020

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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

31 March 2020

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

31 March 2020

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Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

31 March 2020

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Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle

31 March 2020

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Gene therapy and the germline

31 March 2020

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Host and vector-dependent effects on the risk of germline transmission of AAV vectors

31 March 2020

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Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial

31 March 2020

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Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

31 March 2020

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Adeno-associated virus vectors can be efficiently produced without helper virus

31 March 2020

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

31 March 2020

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AAV serotype 2 vectors preferentially integrate into active genes in mice

31 March 2020

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Chromosomal effects of adeno-associated virus vector integration

31 March 2020

1 reference

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

31 March 2020

1 reference

Current status of gene therapy for inherited lung diseases

31 March 2020

1 reference

Clinical gene transfer studies for hemophilia A.

31 March 2020

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Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates

31 March 2020

1 reference

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

31 March 2020

1 reference

Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.

31 March 2020

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Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

31 March 2020

1 reference

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

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Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

31 March 2020

1 reference

Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial.

31 March 2020

1 reference

Investigation of the cause of death in a gene-therapy trial

31 March 2020

1 reference

Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor alpha antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study.

31 March 2020

1 reference

Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 Study

31 March 2020

1 reference

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery

31 March 2020

1 reference

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

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Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

31 March 2020

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Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice

31 March 2020

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

31 March 2020

1 reference

Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system

31 March 2020

1 reference

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

31 March 2020

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

31 March 2020

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Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia

31 March 2020

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Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy.

31 March 2020

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12 December 2020

A coagulation factor IX-deficient mouse model for human hemophilia B.

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12 December 2020

Nucleotide sequence and organization of the adeno-associated virus 2 genome

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12 December 2020

CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease.

1 reference

12 December 2020

A deletion mutation causes hemophilia B in Lhasa Apso dogs

1 reference

12 December 2020

Gene therapy restores vision in a canine model of childhood blindness

1 reference

12 December 2020

Vision 1 year after gene therapy for Leber's congenital amaurosis

1 reference

12 December 2020

Identifiers

DOI

10.1038/NRG2988

1 reference

24 August 2017

1 reference

PubMed ID

21499295