(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

5

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24 August 2017

341-355

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

24 August 2017

0 references

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Effect of gene therapy on visual function in Leber's congenital amaurosis

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Hemophilia gene therapy: a Holy Grail found

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Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

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Leber congenital amaurosis: genes, proteins and disease mechanisms

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Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

31 March 2020

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Case 15-2009: a man with coma after cardiac arrest

31 March 2020

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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

31 March 2020

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Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

31 March 2020

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Newborn screening for treatable genetic conditions: past, present and future

31 March 2020

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Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1.

31 March 2020

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Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus

31 March 2020

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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.

31 March 2020

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Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction

31 March 2020

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Haemophilia prophylaxis in young patients--a long-term follow-up

31 March 2020

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Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia

31 March 2020

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Comprehensive care for haemophilia around the world

31 March 2020

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A factor IX-deficient mouse model for hemophilia B gene therapy

31 March 2020

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Expression of human factor IX in mice after injection of genetically modified myoblasts

31 March 2020

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Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A.

31 March 2020

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Canine hemophilia B resulting from a point mutation with unusual consequences

31 March 2020

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Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes

31 March 2020

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

31 March 2020

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

31 March 2020

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Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

31 March 2020

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

31 March 2020

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

31 March 2020

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Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

31 March 2020

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AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

31 March 2020

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Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

31 March 2020

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Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice

31 March 2020

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

31 March 2020

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

31 March 2020

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Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

31 March 2020

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Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

31 March 2020

1 reference

Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses

31 March 2020

1 reference

Cellular immune response to cryptic epitopes during therapeutic gene transfer

31 March 2020

1 reference

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

31 March 2020

1 reference

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

31 March 2020

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

31 March 2020

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AAV vector integration sites in mouse hepatocellular carcinoma.

31 March 2020

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Assessing the potential for AAV vector genotoxicity in a murine model

31 March 2020

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

31 March 2020

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Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

31 March 2020

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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

31 March 2020

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Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

31 March 2020

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Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

31 March 2020

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AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

31 March 2020

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Current prospects for RNA interference-based therapies

31 March 2020

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Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

31 March 2020

1 reference

Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease

31 March 2020

1 reference

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

31 March 2020

1 reference

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

31 March 2020

1 reference

Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).

31 March 2020

1 reference

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

31 March 2020

1 reference

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

31 March 2020

1 reference

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

31 March 2020

1 reference

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

31 March 2020

1 reference

Convection-enhanced delivery of macromolecules in the brain

31 March 2020

1 reference

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

31 March 2020

1 reference

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

31 March 2020

1 reference

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

31 March 2020

1 reference

Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.

31 March 2020

1 reference

Risk factors for hemorrhage during microelectrode-guided deep brain stimulator implantation for movement disorders

31 March 2020

1 reference

Aspartoacylase deficiency and N-acetylaspartic aciduria in patients with canavan disease

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

31 March 2020

1 reference

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

31 March 2020

1 reference

Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

31 March 2020

1 reference

CSF biomarkers and incipient Alzheimer disease in patients with mild cognitive impairment.

31 March 2020

1 reference

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

31 March 2020

1 reference

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

31 March 2020

1 reference

Dystrophin immunity in Duchenne's muscular dystrophy.

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

The danger model: a renewed sense of self

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Gene therapy and the germline

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

AAV serotype 2 vectors preferentially integrate into active genes in mice

31 March 2020

1 reference

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

1 reference

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

1 reference

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

1 reference

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

1 reference

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

31 March 2020

1 reference

Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice

31 March 2020

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

12 December 2020

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

1 reference

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