AAV4Cancer
Terapias Avanzadas para Enfermedades Hepáticas Raras
Publications (18) Publications in which a researcher has participated
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Hepatology
2024
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Deciphering the Role of Post-Translational Modifications and Cellular Location of Hepatitis Delta Virus (HDV) Antigens in HDV-Mediated Liver Damage in Mice
Viruses, Vol. 16, Núm. 3
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Gene editing in liver diseases
FEBS Letters, Vol. 598, Núm. 19, pp. 2348-2371
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Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation
Nature Communications, Vol. 15, Núm. 1
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In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells
International Journal of Molecular Sciences, Vol. 25, Núm. 23
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Protective role of RIPK1 scaffolding against HDV-induced hepatocyte cell death and the significance of cytokines in mice
PLoS Pathogens, Vol. 20, Núm. 5
2023
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Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors
Human gene therapy, Vol. 34, Núm. 7-8, pp. 273-288
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Exogenous aralar/slc25a12 can replace citrin/slc25a13 as malate aspartate shuttle component in liver
Molecular Genetics and Metabolism Reports, Vol. 35
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Gene therapy for liver diseases — progress and challenges
Nature Reviews Gastroenterology and Hepatology, Vol. 20, Núm. 5, pp. 288-305
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Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells
Pharmaceutics, Vol. 15, Núm. 10
2022
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Gene Therapy for Acquired and Genetic Cholestasis
Biomedicines, Vol. 10, Núm. 6
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Inspecting the Ribozyme Region of Hepatitis Delta Virus Genotype 1: Conservation and Variability
Viruses, Vol. 14, Núm. 2
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mRNA and gene editing: Late breaking therapies in liver diseases
Hepatology, Vol. 76, Núm. 3, pp. 869-887
2021
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Linking the Expression of Therapeutic Genes to Unfolded Protein Response: A New Option for Anti-Hepatitis B Virus Gene Therapy
Human Gene Therapy, Vol. 32, Núm. 7-8, pp. 341-348
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
2019
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Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
Nature Communications, Vol. 10, Núm. 1
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Genetic-Based Approaches to Inherited Metabolic Liver Diseases
Human Gene Therapy, Vol. 30, Núm. 10, pp. 1190-1203
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Liver Expression of a MiniATP7B Gene Results in Long-Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice
Hepatology, Vol. 70, Núm. 1, pp. 108-126