A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
about
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.
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
label
A translationally optimized AA ...... on of Crigler-Najjar syndrome.
@en
A translationally optimized AA ...... on of Crigler-Najjar syndrome.
@nl
prefLabel
A translationally optimized AA ...... on of Crigler-Najjar syndrome.
@en
A translationally optimized AA ...... on of Crigler-Najjar syndrome.
@nl
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P2860
P356
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A translationally optimized AA ...... on of Crigler-Najjar syndrome.
@en
P2093
Alban Vignaud
Bernard Gjata
Christian Leborgne
Fanny Collaud
Federico Mingozzi
Giulia Bortolussi
Giuseppe Ronzitti
Laetitia van Wittenberghe
Marcelo Simon Sola
Patrice Vidal
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P356
10.1038/MTM.2016.49
P577
2016-07-20T00:00:00Z