Terapias Avanzadas para Enfermedades Hepáticas Raras
AAV4Cancer
Centro de Investigación Médica Aplicada
Pamplona, EspañaPublicaciones en colaboración con investigadores/as de Centro de Investigación Médica Aplicada (141)
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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Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1
EMBO molecular medicine, Vol. 16, Núm. 1, pp. 112-131
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GCN2 inhibition reduces mutant SOD1 clustering and toxicity and delays disease progression in an amyotrophic lateral sclerosis mouse model
Translational Neurodegeneration
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Gene editing in liver diseases
FEBS Letters
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Profile of plasma microRNAs as a potential biomarker of Wilson’s disease
Journal of Gastroenterology, Vol. 59, Núm. 10, pp. 921-931
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Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy
Human gene therapy, Vol. 35, Núm. 17-18, pp. 582
2023
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AG5 is a potent non-steroidal anti-inflammatory and immune regulator that preserves innate immunity
Biomedicine and Pharmacotherapy, Vol. 169
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Cardiac Progenitor Cell Exosomal miR-935 Protects against Oxidative Stress
Cells, Vol. 12, Núm. 18
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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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Microglia and astrocyte activation is region-dependent in the α-synuclein mouse model of Parkinson's disease
GLIA, Vol. 71, Núm. 3, pp. 571-587
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Photodynamic nasal SARS-CoV-2 decolonization shortens infectivity and influences specific T-Cell responses
Frontiers in Cellular and Infection Microbiology, Vol. 13
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Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy
JHEP Reports, Vol. 5, Núm. 5
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Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV)
Bone Marrow Transplantation
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Stabilization of 14-3-3 protein-protein interactions with Fusicoccin-A decreases alpha-synuclein dependent cell-autonomous death in neuronal and mouse models
Neurobiology of Disease, Vol. 183
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Treatment with the senolytics dasatinib/quercetin reduces SARS-CoV-2-related mortality in mice
Aging Cell, Vol. 22, Núm. 3
2022
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A minimal bile salt excretory pump promoter allows bile acid-driven physiological regulation of transgene expression from a gene therapy vector
Cell and Bioscience, Vol. 12, Núm. 1
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Comment on “Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4-/- mouse model of PFIC3”
Journal of Hepatology
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Gene Therapy for Acquired and Genetic Cholestasis
Biomedicines, Vol. 10, Núm. 6
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High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease
Molecular Therapy - Methods and Clinical Development, Vol. 26, pp. 98-106
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Inspecting the Ribozyme Region of Hepatitis Delta Virus Genotype 1: Conservation and Variability
Viruses, Vol. 14, Núm. 2