A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. - Wikidata - wikimedia - TriplyDB

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

http://www.wikidata.org/entity/Q37313599

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Unraveling the complex story of immune responses to AAV vectors trial after trial.Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I.Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.Emerging Issues in AAV-Mediated In Vivo Gene Therapy.Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

http://www.wikidata.org/entity/Q37313599

description

article científic

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article scientifique

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articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on 20 July 2016

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vedecký článok

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vetenskaplig artikel

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videnskabelig artikel

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vědecký článek

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name

A translationally optimized AA ...... on of Crigler-Najjar syndrome.

@en

A translationally optimized AA ...... on of Crigler-Najjar syndrome.

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type

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A translationally optimized AA ...... on of Crigler-Najjar syndrome.

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A translationally optimized AA ...... on of Crigler-Najjar syndrome.

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prefLabel

A translationally optimized AA ...... on of Crigler-Najjar syndrome.

@en

A translationally optimized AA ...... on of Crigler-Najjar syndrome.

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Molecular Therapy. Methods & Clinical Development

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A translationally optimized AA ...... on of Crigler-Najjar syndrome.

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Alban Vignaud

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Bernard Gjata

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Christian Leborgne

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Fanny Collaud

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Federico Mingozzi

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Giulia Bortolussi

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Giuseppe Ronzitti

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Laetitia van Wittenberghe

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Marcelo Simon Sola

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Patrice Vidal

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Genetic lesions of bilirubin uridine-diphosphoglucuronate glucuronosyltransferase (UGT1A1) causing Crigler-Najjar and Gilbert syndromes: correlation of genotype to phenotype

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation

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16049

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scholarly article

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10.1038/MTM.2016.49

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3

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Andrés F. Muro

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2016-07-20T00:00:00Z

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27722180

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5052023

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