Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy-Reference-Cited by-同舟云学术

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Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy

Published:2012-10 Issue:10 Volume:20 Page:1844-1851
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Lee Eun K.,Hu Chuhong,Bhargava Ragini,Rozengurt Nora,Stout David,Grody Wayne W,Cederbaum Stephen D,Lipshutz Gerald S

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference25 articles.

1. The human arginases and arginase deficiency;Iyer;J Inherit Metab Dis,1998

2. Arginase I deficiency: severe infantile presentation with hyperammonemia: more common than reported;Jain-Ghai;Mol Genet Metab,2011

3. Arginase deficiency with lethal neonatal expression: evidence for the glutamine hypothesis of cerebral edema;Picker;J Pediatr,2003

4. Argininemia: a treatable genetic cause of progressive spastic diplegia simulating cerebral palsy: case reports and literature review;Prasad;J Child Neurol,1997

5. Diffusion tensor imaging in arginase deficiency reveals damage to corticospinal tracts;Oldham;Pediatr Neurol,2010

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1. Liver gene transfer for metabolite detoxification in inherited metabolic diseases;FEBS Letters;2024-06-17

2. Plasma arginine levels in arginase deficiency in the “real world”;Molecular Genetics and Metabolism Reports;2024-03

3. Gene therapy for glycogen storage diseases;Journal of Inherited Metabolic Disease;2023-07-27

4. Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia;Molecular Therapy - Methods & Clinical Development;2023-06

5. Gene therapy for urea cycle defects: An update from historical perspectives to future prospects;Journal of Inherited Metabolic Disease;2023-04-18

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