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Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

Proceedings of the National Academy of Sciences - PNAS, 2019-10, Vol.116 (42), p.21150-21159

2019

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Newborn screening for hyperargininemia due to arginase 1 deficiency

Molecular genetics and metabolism, 2017-08, Vol.121 (4), p.308-313

2017

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Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency

Molecular therapy. Nucleic acids, 2022-06, Vol.28, p.859-874

2022

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Is it time to retire fragile X testing as a first-tier test for developmental delay, intellectual disability, and autism spectrum disorder?

Genetics in medicine, 2017-12, Vol.19 (12), p.1380-1380

2017

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Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice

Molecular genetics and metabolism, 2018-06, Vol.124 (2), p.114-123

2018

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The role and control of arginine levels in arginase 1 deficiency

Journal of inherited metabolic disease, 2023-01, Vol.46 (1), p.3-14

2023

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Contrasting features of urea cycle disorders in human patients and knockout mouse models

Molecular genetics and metabolism, 2008-01, Vol.93 (1), p.7-14

2008

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Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders

Molecular genetics and metabolism, 2023-04, Vol.138 (4), p.107558-107558, Article 107558

2023

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Confidential genetic testing and electronic health records: A survey of current practices among Huntington disease testing centers

Molecular genetics & genomic medicine, 2020-01, Vol.8 (1), p.e1026-n/a

2020

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Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells

Molecular therapy. Nucleic acids, 2016, Vol.5 (11), p.e394-e394, Article e394

2016

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Mouse Model for Human Arginase Deficiency

Molecular and Cellular Biology, 2002-07, Vol.22 (13), p.4491-4498

2002

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l-Citrulline Protects from Kidney Damage in Type 1 Diabetic Mice

Frontiers in immunology, 2013-01, Vol.4, p.480

2013

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Arginase 1: an unexpected mediator of pulmonary capillary barrier dysfunction in models of acute lung injury

Frontiers in immunology, 2013-01, Vol.4, p.228

2013

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Diffusion Tensor Imaging in Arginase Deficiency Reveals Damage to Corticospinal Tracts

Pediatric neurology, 2010, Vol.42 (1), p.49-52

2010

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Increased plasma and tissue guanidino compounds in a mouse model of hyperargininemia

Molecular genetics and metabolism, 2008-02, Vol.93 (2), p.172-178

2008

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Biopsy-proven Hepatocellular Carcinoma in a 53-year-old Woman With Arginase Deficiency

Pediatric and developmental pathology, 2017-12, Vol.20 (6), p.517-521

2017

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Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy

The Journal of neuroscience, 2016-06, Vol.36 (25), p.6680-6690

2016

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Cloning and Characterization of Human Agmatinase

Molecular genetics and metabolism, 2002-03, Vol.75 (3), p.209-218

2002

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Diabetes-induced vascular dysfunction involves arginase I

American journal of physiology. Heart and circulatory physiology, 2012-01, Vol.302 (1), p.H159-H166

2012

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Cross-sectional multicenter study of patients with urea cycle disorders in the United States

Molecular genetics and metabolism, 2008-08, Vol.94 (4), p.397-402

2008

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Widespread Expression of Arginase I in Mouse Tissues: Biochemical and Physiological Implications

The journal of histochemistry and cytochemistry, 2003-09, Vol.51 (9), p.1151-1160

2003

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Polyamine homeostasis in arginase knockout mice

American Journal of Physiology: Cell Physiology, 2007-10, Vol.293 (4), p.C1296-C1301

2007

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

Molecular therapy, 2012-10, Vol.20 (10), p.1844-1851

2012

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Hyperargininemia due to liver arginase deficiency

Molecular genetics and metabolism, 2005-03, Vol.84 (3), p.243-251

2005

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Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

Newborn screening for hyperargininemia due to arginase 1 deficiency

Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency

Is it time to retire fragile X testing as a first-tier test for developmental delay, intellectual disability, and autism spectrum disorder?

Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice

The role and control of arginine levels in arginase 1 deficiency

Contrasting features of urea cycle disorders in human patients and knockout mouse models

Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders

Confidential genetic testing and electronic health records: A survey of current practices among Huntington disease testing centers

Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells

Mouse Model for Human Arginase Deficiency

l-Citrulline Protects from Kidney Damage in Type 1 Diabetic Mice

Arginase 1: an unexpected mediator of pulmonary capillary barrier dysfunction in models of acute lung injury

Diffusion Tensor Imaging in Arginase Deficiency Reveals Damage to Corticospinal Tracts

Secondary ACMG and non-ACMG genetic findings in a multiethnic cohort of 16,713 pediatric participants

Increased plasma and tissue guanidino compounds in a mouse model of hyperargininemia

Biopsy-proven Hepatocellular Carcinoma in a 53-year-old Woman With Arginase Deficiency

Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy

Cloning and Characterization of Human Agmatinase

Diabetes-induced vascular dysfunction involves arginase I

Cross-sectional multicenter study of patients with urea cycle disorders in the United States

Widespread Expression of Arginase I in Mouse Tissues: Biochemical and Physiological Implications

Polyamine homeostasis in arginase knockout mice

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

Hyperargininemia due to liver arginase deficiency

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