Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. - Wikidata - wikimedia - TriplyDB

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

http://www.wikidata.org/entity/Q35105022

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Multi-OMICs and Genome Editing Perspectives on Liver Cancer Signaling Networks Adeno-associated Virus as a Mammalian DNA Vector Liver-targeted gene therapy: Approaches and challenges A universal system to select gene-modified hepatocytes in vivo Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo CRISPR/Cas9 - Mediated Precise Targeted Integration In Vivo Using a Double Cut Donor with Short Homology Arms.Fumarylacetoacetate hydrolase deficient pigs are a novel large animal model of metabolic liver disease Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.rAAV-mediated tumorigenesis: still unresolved after an AAV assault Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.Perinatal gene transfer to the liver.Efficient production of Fah-null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer.AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo Induction of hepatocellular carcinoma by in vivo gene targeting.The threefold protrusions of adeno-associated virus type 8 are involved in cell surface targeting as well as postattachment processing.AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence.In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs.CRISPR Editing in Biological and Biomedical Investigation.Viral vectors for therapy of neurologic diseases.Gene editing technology as an approach to the treatment of liver diseases.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Current and future prospects for hemophilia gene therapy.Ifit1 Protects Against Lipopolysaccharide and D-galactosamine-Induced Fatal Hepatitis by Inhibiting Activation of the JNK Pathway.CRISPR/Cas9-mediated correction of human genetic disease.Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications.Gene Therapy in Tyrosinemia: Potential and Pitfalls.Fah Knockout Animals as Models for Therapeutic Liver Repopulation.Efficient liver gene transfer with foamy virus vectors.Construction and application of a novel hepatocyte-directed vector to simultaneous knockdown and overexpression of multiple genes.Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.Assembly of CRISPR ribonucleoproteins with biotinylated oligonucleotides via an RNA aptamer for precise gene editing.Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.Cas9-nickase-mediated genome editing corrects hereditary tyrosinemia in rats.CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy.Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40.Homologous recombination mediates stable Fah gene integration and phenotypic correction in tyrosinaemia mouse-model.All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo

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P2860

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

http://www.wikidata.org/entity/Q35105022

description

2010 nî lūn-bûn

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2010 թուականի Ապրիլին հրատարակուած գիտական յօդուած

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2010 թվականի ապրիլին հրատարակված գիտական հոդված

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2010年の論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年论文

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... ereditary tyrosinemia in vivo.

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Adeno-associated virus gene re ...... hereditary tyrosinemia in vivo

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Karsten Wursthorn

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Mark A Kay

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Milton J Finegold

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P2860

Point mutations in the murine fumarylacetoacetate hydrolase gene: Animal models for the human genetic disorder hereditary tyrosinemia type 1

High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors.

Adeno-associated virus vectors and hematology

Gene therapy for the hemophilias.

Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver.

Increased efficiency of oligonucleotide-mediated gene repair through slowing replication fork progression.

Chromosomal integration and homologous gene targeting by replication-incompetent vectors based on the autonomous parvovirus minute virus of mice.

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

The pathophysiology and treatment of hereditary tyrosinemia type 1.

Correction of the UDP-glucuronosyltransferase gene defect in the gunn rat model of crigler-najjar syndrome type I with a chimeric oligonucleotide

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1200-1208

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Nicole K. Paulk

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20162619

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3136243

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