Hepatología: porfirias y carcinogénesis
Centro de Investigación Médica Aplicada
Pamplona, EspañaPublications in collaboration with researchers from Centro de Investigación Médica Aplicada (35)
2024
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Exploring current and emerging therapies for porphyrias
Liver International, Vol. 44, Núm. 9, pp. 2174-2190
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Porphyrins and the porphyrias
Liver International
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Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates
Gut
2023
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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, Vol. 24, Núm. 15
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Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria
International Journal of Molecular Sciences, Vol. 24, Núm. 1
2022
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Mechanistic modelling of enzyme-restoration effects of new recombinant liver-targeted proteins in acute intermittent porphyria
British Journal of Pharmacology, Vol. 179, Núm. 14, pp. 3815-3830
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Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases
International Review of Cell and Molecular Biology (Elsevier Inc.), pp. 55-96
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Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment
Life, Vol. 12, Núm. 11
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Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria
Science Translational Medicine, Vol. 14, Núm. 627
2021
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Acute intermittent porphyria, givosiran, and homocysteine
Journal of Inherited Metabolic Disease
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Diagnosis and management of inborn errors of metabolism in adult patients in the emergency department
Diagnostics, Vol. 11, Núm. 11
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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, Vol. 25, pp. 207-219
2020
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Brain ventricular enlargement in human and murine acute intermittent porphyria
Human molecular genetics, Vol. 29, Núm. 19, pp. 3211-3223
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Disease pharmacokinetic–pharmacodynamic modelling in acute intermittent porphyria to support the development of mRNA-based therapies
British Journal of Pharmacology, Vol. 177, Núm. 14, pp. 3168-3182
2019
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Messenger RNA therapy for rare genetic metabolic diseases
Gut, Vol. 68, Núm. 7, pp. 1323-1330
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Non-parenchymal TREM-2 protects the liver from immune-mediated hepatocellular damage
Gut, Vol. 68, Núm. 3, pp. 533-546
2018
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An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria
Human Gene Therapy, Vol. 29, Núm. 4, pp. 480-491
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Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Nature Medicine, Vol. 24, Núm. 12, pp. 1899-1909
2016
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Glucose metabolism during fasting is altered in experimental porphobilinogen deaminase deficiency
Human Molecular Genetics, Vol. 25, Núm. 7, pp. 1318-1327
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Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Journal of Hepatology, Vol. 65, Núm. 4, pp. 776-783