(Q33902499)

24386892

29 July 2017

title

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

1 reference

29 July 2017

1

1 reference

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

13 June 2020

author name string

Li Zhong

series ordinal

2

1 reference

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

13 June 2020

M Abu Nahid

series ordinal

3

1 reference

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

13 June 2020

Guangping Gao

series ordinal

4

1 reference

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

13 June 2020

language of work or name

English

0 references

publication date

3 January 2014

1 reference

24386892

Expert Opinion on Drug Delivery

29 July 2017

published in

1 reference

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

13 June 2020

volume

11

1 reference

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

13 June 2020

issue

3

1 reference

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

13 June 2020

page(s)

345-364

1 reference

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

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

13 June 2020

cites work

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5 July 2018

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5 July 2018

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

5 July 2018

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5 July 2018

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5 July 2018

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A largely random AAV integration profile after LPLD gene therapy

5 July 2018

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Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease

5 July 2018

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5 July 2018

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Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

5 July 2018

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Therapeutic cardiac-targeted delivery of miR-1 reverses pressure overload-induced cardiac hypertrophy and attenuates pathological remodeling

5 July 2018

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5 July 2018

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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

5 July 2018

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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

5 July 2018

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

5 July 2018

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

5 July 2018

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

5 July 2018

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

5 July 2018

1 reference

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

5 July 2018

1 reference

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

5 July 2018

1 reference

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

5 July 2018

1 reference

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

5 July 2018

1 reference

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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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Antibody gene transfer for HIV immunoprophylaxis

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

5 July 2018

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Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

5 July 2018

1 reference

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Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies

5 July 2018

1 reference

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Gene therapy for muscular dystrophy: lessons learned and path forward

5 July 2018

1 reference

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Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

5 July 2018

1 reference

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Adeno-associated virus vectorology, manufacturing, and clinical applications

5 July 2018

1 reference

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AAV6.βARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model

5 July 2018

1 reference

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Concomitant intravenous nitroglycerin with intracoronary delivery of AAV1.SERCA2a enhances gene transfer in porcine hearts

5 July 2018

1 reference

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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

5 July 2018

1 reference

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

Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping

5 July 2018

1 reference

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

AAV6-βARKct gene delivery mediated by molecular cardiac surgery with recirculating delivery (MCARD) in sheep results in robust gene expression and increased adrenergic reserve

5 July 2018

1 reference

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

Antibody-based protection against HIV infection by vectored immunoprophylaxis

5 July 2018

1 reference

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

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

5 July 2018

1 reference

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

Improved function of the failing rat heart by regulated expression of insulin-like growth factor I via intramuscular gene transfer

5 July 2018

1 reference

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

Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials

5 July 2018

1 reference

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

Innate Immune Responses to AAV Vectors

5 July 2018

1 reference

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

Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

5 July 2018

1 reference

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5 July 2018

1 reference

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Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.

5 July 2018

1 reference

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AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

5 July 2018

1 reference

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Gene therapy for haemophilia: a long and winding road.

5 July 2018

1 reference

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Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.

5 July 2018

1 reference

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Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+-ATPase in patients with advanced heart failure

5 July 2018

1 reference

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A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.

5 July 2018

1 reference

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

5 July 2018

1 reference

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Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines

5 July 2018

1 reference

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Pompe disease gene therapy.

5 July 2018

1 reference

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

5 July 2018

1 reference

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Can muscle regeneration fail in chronic inflammation: a weakness in inflammatory myopathies?

5 July 2018

1 reference

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

Directed evolution of adeno-associated virus (AAV) as vector for muscle gene therapy

5 July 2018

1 reference

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A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes

5 July 2018

1 reference

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MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression

5 July 2018

1 reference

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

The complex and evolving story of T cell activation to AAV vector-encoded transgene products

5 July 2018

1 reference

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

Physiological and tissue-specific vectors for treatment of inherited diseases

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Gene therapy for muscle disease

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Characterization of genome integrity for oversized recombinant AAV vector

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Immune responses to AAV in clinical trials

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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.

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Identification of tissue-specific microRNAs from mouse

5 July 2018

1 reference

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

Cross-presentation in viral immunity and self-tolerance.

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Immunological capabilities of skeletal muscle cells

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Antigen processing and presentation by a murine myoblast cell line

5 July 2018

1 reference

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

The immunobiology of muscle

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Isolation of adenovirus-associated viruses from man.

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

Stimulation of fetal hemoglobin synthesis by erythropoietin in baboons

5 July 2018

1 reference

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

Site-specific integration by adeno-associated virus

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

5 July 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Gene therapy on demand: site specific regulation of gene therapy

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

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

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.

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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/10.1517%2F17425247.2014.871258

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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/10.1517%2F17425247.2014.871258

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

Gene transfer into skeletal muscle using novel AAV serotypes.

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

21 January 2018

1 reference

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

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

29 October 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Gene therapy for hemophilia

1 reference

12 December 2020

10.1517/17425247.2014.871258

Identifiers

DOI

1 reference

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

13 June 2020

1 reference

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

13 June 2020

24386892

1 reference

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