Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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Delivery of glutamine synthetase gene by baculovirus vectors: a proof of concept for the treatment of acute hyperammonemia.Liver transplantation for urea cycle disorders in pediatric patients: a single-center experience.

P2860

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P2860

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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http://www.wikidata.org/entity/Q45877752

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2012 nî lūn-bûn

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2012年の論文

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2012年学术文章

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2012年学术文章

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2012年学术文章

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2012年学术文章

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2012年学术文章

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2012年學術文章

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2012年學術文章

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2012年學術文章

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name

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

@en

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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type

Item

label

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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prefLabel

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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P1476

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders

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P2093

Allison P Dane

http://www.w3.org/2001/XMLSchema#string

Sharon C Cunningham

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P2860

Vision 1 year after gene therapy for Leber's congenital amaurosis

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

Effect of gene therapy on visual function in Leber's congenital amaurosis

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Stem-cell gene therapy for the Wiskott-Aldrich syndrome.

AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.

P2888

s10545-012-9467-0

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641-645

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scholarly article

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10.1007/S10545-012-9467-0

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Ian E. Alexander

Cindy Kok

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2012-03-09T00:00:00Z

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221688948

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1025049746

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22403018

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P921

gene therapy

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

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P2860

P2860

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.

description

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P1433

P1476

P2093

P2860

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