Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors.
about
Progresses towards safe and efficient gene therapy vectorsStrategies to rescue the consequences of inducible arginase-1 deficiency in micePreclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type miceLong-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.Kinetics of adeno-associated virus serotype 2 (AAV2) and AAV8 capsid antigen presentation in vivo are identical.Emerging therapies for mitochondrial disorders.Cocaine hydrolase gene therapy for cocaine abuseAAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
P2860
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P2860
Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors.
description
2011 nî lūn-bûn
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2011年の論文
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2011年学术文章
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2011年学术文章
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name
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@ast
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@en
type
label
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@ast
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@en
prefLabel
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@ast
Sustained correction of OTC de ...... -complementary AAV2/8 vectors.
@en
P2093
P2860
P356
P1433
P1476
Sustained correction of OTC de ...... f-complementary AAV2/8 vectors
@en
P2093
D McMenamin
M L Batshaw
P2860
P2888
P304
P356
10.1038/GT.2011.111
P577
2011-08-18T00:00:00Z