The potential of adeno-associated viral vectors for gene delivery to muscle tissue (Q33902499)

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Language	Label	Description	Also known as
English	The potential of adeno-associated viral vectors for gene delivery to muscle tissue	scientific article

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scholarly article

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Europe PubMed Central

PubMed publication ID

29 July 2017

The potential of adeno-associated viral vectors for gene delivery to muscle tissue (English)

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Europe PubMed Central

PubMed publication ID

29 July 2017

1

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13 June 2020

author name string

Li Zhong

2

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13 June 2020

M Abu Nahid

3

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Europe PubMed Central

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13 June 2020

Guangping Gao

4

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13 June 2020

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3 January 2014

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Europe PubMed Central

PubMed publication ID

29 July 2017

Expert Opinion on Drug Delivery

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13 June 2020

11

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13 June 2020

3

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13 June 2020

345-364

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13 June 2020

Intravenous adeno-associated virus serotype 8 encoding urocortin-2 provides sustained augmentation of left ventricular function in mice

1 reference

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Restriction of calpain3 expression to the skeletal muscle prevents cardiac toxicity and corrects pathology in a murine model of limb-girdle muscular dystrophy

1 reference

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Overcoming preexisting humoral immunity to AAV using capsid decoys

1 reference

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Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions

1 reference

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CpG-depleted adeno-associated virus vectors evade immune detection.

1 reference

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

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Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes

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Cardiac-selective expression of extracellular superoxide dismutase after systemic injection of adeno-associated virus 9 protects the heart against post-myocardial infarction left ventricular remodeling

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Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery.

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Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

1 reference

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Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking

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Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus

1 reference

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Adeno-associated viral-mediated LARGE gene therapy rescues the muscular dystrophic phenotype in mouse models of dystroglycanopathy

1 reference

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Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

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Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

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Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

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AAV9.I-1c delivered via direct coronary infusion in a porcine model of heart failure improves contractility and mitigates adverse remodeling

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Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia

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Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors.

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Adeno-associated virus serotype 8 (AAV8) delivery of recombinant A20 to skeletal muscle reduces pathological activation of nuclear factor (NF)-κB in muscle of mdx mice

1 reference

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Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

1 reference

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Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer

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Pompe disease gene therapy.

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Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.

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5 July 2018

Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease

1 reference

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Gene delivery of sarcoplasmic reticulum calcium ATPase inhibits ventricular remodeling in ischemic mitral regurgitation

1 reference

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5 July 2018

Gene therapy for muscle disease

1 reference

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5 July 2018

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs

1 reference

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5 July 2018

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

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5 July 2018

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

1 reference

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5 July 2018

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

1 reference

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5 July 2018

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

1 reference

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5 July 2018

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

1 reference

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5 July 2018

Characterization of genome integrity for oversized recombinant AAV vector

1 reference

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5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Myocardial adeno-associated virus serotype 6-betaARKct gene therapy improves cardiac function and normalizes the neurohormonal axis in chronic heart failure

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Hydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growth

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr-/- -dependent allergy mouse model

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Adeno-associated virus type 12 (AAV12): a novel AAV serotype with sialic acid- and heparan sulfate proteoglycan-independent transduction activity

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Immune responses to AAV in clinical trials

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Economized large-scale production of high yield of rAAV for gene therapy applications exploiting baculovirus expression system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Stable myocardial-specific AAV6-S100A1 gene therapy results in chronic functional heart failure rescue

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Efficient whole-body transduction with trans-splicing adeno-associated viral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

PEG conjugation moderately protects adeno-associated viral vectors against antibody neutralization

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Generation of neutralizing activity against human immunodeficiency virus type 1 in serum by antibody gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Identification of tissue-specific microRNAs from mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Cross-presentation in viral immunity and self-tolerance.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Immunological capabilities of skeletal muscle cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Immune responses to adenovirus and adeno-associated virus in humans.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Long-term regulated expression of growth hormone in mice after intramuscular gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Ecdysone-inducible gene expression in mammalian cells and transgenic mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Antigen processing and presentation by a murine myoblast cell line

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

The immunobiology of muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Herpes Simplex Virus Types 1 and 2 Completely Help Adenovirus-Associated Virus Replication

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Isolation of adenovirus-associated viruses from man.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Vaccinia virus, herpes simplex virus, and carcinogens induce DNA amplification in a human cell line and support replication of a helpervirus dependent parvovirus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Stimulation of fetal hemoglobin synthesis by erythropoietin in baboons

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Site-specific integration by adeno-associated virus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Coculture with autologous myotubes of cytotoxic T cells isolated from muscle in inflammatory myopathies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian Cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

5 July 2018

Exploiting natural diversity of AAV for the design of vectors with novel properties

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Transendocardial delivery of AAV6 results in highly efficient and global cardiac gene transfer in rhesus macaques

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Prevention of cardiomyopathy in delta-sarcoglycan knockout mice after systemic transfer of targeted adeno-associated viral vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Gene therapy on demand: site specific regulation of gene therapy

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Tetracycline-inducible interleukin-10 gene transfer mediated by an adeno-associated virus: application to experimental arthritis

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Dystrophin immunity in Duchenne's muscular dystrophy.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

AAV-mediated gene therapy to the isolated limb in rhesus macaques

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Differential myocardial gene delivery by recombinant serotype-specific adeno-associated viral vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

IGF1 gene transfer into skeletal muscle using recombinant adeno-associated virus in a rat model of liver cirrhosis.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Efficient and long-term intracardiac gene transfer in delta-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

PDGF-A, -C, and -D but not PDGF-B increase TGF-beta1 and chronic rejection in rat cardiac allografts.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Rescue of dystrophic skeletal muscle by PGC-1α involves restored expression of dystrophin-associated protein complex components and satellite cell signaling.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Gene transfer into skeletal muscle using novel AAV serotypes.

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Adenovirus and adeno-associated virus-mediated delivery of human myophosphorylase cDNA and LacZ cDNA to muscle in the ovine model of McArdle's disease: expression and re-expression of glycogen phosphorylase

1 reference

https://api.crossref.org/works/10.1517%2F17425247.2014.871258

21 January 2018

Retrovirus-mediated transfer of the erythropoietin gene in hematopoietic cells improves the erythrocyte phenotype in murine beta-thalassemia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4098646

29 October 2018

The gene therapy journey for hemophilia: are we there yet?

1 reference

https://pubmed.ncbi.nlm.nih.gov/24386892

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Intraperitoneal AAV9-shRNA inhibits target expression in neonatal skeletal and cardiac muscles

1 reference

https://pubmed.ncbi.nlm.nih.gov/24386892

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector

1 reference

https://pubmed.ncbi.nlm.nih.gov/24386892

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy for hemophilia

1 reference

https://pubmed.ncbi.nlm.nih.gov/24386892

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1517/17425247.2014.871258

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24386892%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24386892%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24386892%20AND%20SRC:MED&resulttype=core&format=json

13 June 2020

ResearchGate publication ID

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