Ana
Sampedro Pascual
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Instituto de Salud Carlos III
Madrid, EspañaInstituto de Salud Carlos III -ko ikertzaileekin lankidetzan egindako argitalpenak (4)
2022
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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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High prevalence of insulin resistance in asymptomatic patients with acute intermittent porphyria and liver-targeted insulin as a novel therapeutic approach
Biomedicines, Vol. 9, Núm. 3, pp. 1-18
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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
2018
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Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Nature Medicine, Vol. 24, Núm. 12, pp. 1899-1909