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Messenger RNA therapy for rare genetic metabolic diseases

Gut, 2019-07, Vol.68 (7), p.1323-1330

2019

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Non-parenchymal TREM-2 protects the liver from immune-mediated hepatocellular damage

Gut, 2019-03, Vol.68 (3), p.533-546

2019

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Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria

International journal of molecular sciences, 2022-12, Vol.24 (1), p.51

2022

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An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria

Human gene therapy, 2018-04, Vol.29 (4), p.480-491

2018

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Emerging therapies for acute intermittent porphyria

Expert reviews in molecular medicine, 2016-11, Vol.18, p.e17, Article e17

2016

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Bioengineered PBGD variant improves the therapeutic index of gene therapy vectors for acute intermittent porphyria

Human molecular genetics, 2018-11, Vol.27 (21), p.3688-3696

2018

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Heme oxygenase-1 inducer hemin does not inhibit SARS-CoV-2 virus infection

Biomedicine & pharmacotherapy, 2021-05, Vol.137, p.111384-111384, Article 111384

2021

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Nutritional Interventions with Bacillus coagulans Improved Glucose Metabolism and Hyperinsulinemia in Mice with Acute Intermittent Porphyria

International journal of molecular sciences, 2023-07, Vol.24 (15), p.11938

2023

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Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

Journal of translational medicine, 2012-06, Vol.10 (1), p.122-122, Article 122

2012

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The Alpha-Lipoic Acid Improves Glucose Metabolism and Hyperinsulinemia in Acute Intermittent Porphyria: A Nutritional Concept for the Management of Rare Disorders

Cellular and molecular gastroenterology and hepatology, 2024, Vol.17 (3), p.511-514

2024

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Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders

Human gene therapy, 2009-08, Vol.20 (8), p.908-917

2009

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Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment

Life (Basel, Switzerland), 2022-11, Vol.12 (11), p.1858

2022

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mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks

Molecular therapy. Nucleic acids, 2021-09, Vol.25, p.207-219

2021

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Diagnosis and Management of Inborn Errors of Metabolism in Adult Patients in the Emergency Department

Diagnostics (Basel), 2021-11, Vol.11 (11), p.2148

2021

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Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice

Molecular therapy, 2011-02, Vol.19 (2), p.243-250

2011

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High Prevalence of Insulin Resistance in Asymptomatic Patients with Acute Intermittent Porphyria and Liver-Targeted Insulin as a Novel Therapeutic Approach

Biomedicines, 2021-03, Vol.9 (3), p.255

2021

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Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse

PloS one, 2012-03, Vol.7 (3), p.e32978-e32978

2012

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Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus

PloS one, 2014-01, Vol.9 (1), p.e85432-e85432

2014

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Hydroxymethylbilane synthase (aka porphobilinogen deaminase): A novel metabolic tumor suppressor gene in hepatocellular carcinoma

Journal of hepatology, 2022-10, Vol.77 (4), p.912-914

2022

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Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

Journal of hepatology, 2016-10, Vol.65 (4), p.776-783

2016

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Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Nature medicine, 2018-12, Vol.24 (12), p.1899-1909

2018

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Current and innovative emerging therapies for porphyrias with hepatic involvement

Journal of hepatology, 2019-08, Vol.71 (2), p.422-433

2019

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Porphyrins and the porphyrias

Liver international, 2024-09

2024

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Acute intermittent porphyria, givosiran, and homocysteine

Journal of inherited metabolic disease, 2021-07, Vol.44 (4), p.790-791

2021

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Messenger RNA therapy for rare genetic metabolic diseases

Non-parenchymal TREM-2 protects the liver from immune-mediated hepatocellular damage

Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria

An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria

Emerging therapies for acute intermittent porphyria

Bioengineered PBGD variant improves the therapeutic index of gene therapy vectors for acute intermittent porphyria

Heme oxygenase-1 inducer hemin does not inhibit SARS-CoV-2 virus infection

Nutritional Interventions with Bacillus coagulans Improved Glucose Metabolism and Hyperinsulinemia in Mice with Acute Intermittent Porphyria

Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

The Alpha-Lipoic Acid Improves Glucose Metabolism and Hyperinsulinemia in Acute Intermittent Porphyria: A Nutritional Concept for the Management of Rare Disorders

Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders

Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment

mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks

Diagnosis and Management of Inborn Errors of Metabolism in Adult Patients in the Emergency Department

Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice

High Prevalence of Insulin Resistance in Asymptomatic Patients with Acute Intermittent Porphyria and Liver-Targeted Insulin as a Novel Therapeutic Approach

Renal failure affects the enzymatic activities of the three first steps in hepatic heme biosynthesis in the acute intermittent porphyria mouse

Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus

Hydroxymethylbilane synthase (aka porphobilinogen deaminase): A novel metabolic tumor suppressor gene in hepatocellular carcinoma

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Current and innovative emerging therapies for porphyrias with hepatic involvement

Porphyrins and the porphyrias

Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates

Acute intermittent porphyria, givosiran, and homocysteine

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