AAV4Cancer
Terapias Avanzadas para Enfermedades Hepáticas Raras
Publicaciones (68) Publicaciones en las que ha participado algún/a investigador/a
2024
-
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
-
Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1
EMBO molecular medicine, Vol. 16, Núm. 1, pp. 112-131
-
Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations
Molecular Therapy Nucleic Acids, Vol. 35, Núm. 3
-
GCN2 inhibition reduces mutant SOD1 clustering and toxicity and delays disease progression in an amyotrophic lateral sclerosis mouse model
Translational Neurodegeneration
-
Gene editing in liver diseases
FEBS Letters
-
Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation
Nature Communications, Vol. 15, Núm. 1
-
Profile of plasma microRNAs as a potential biomarker of Wilson’s disease
Journal of Gastroenterology, Vol. 59, Núm. 10, pp. 921-931
-
Protective role of RIPK1 scaffolding against HDV-induced hepatocyte cell death and the significance of cytokines in mice
PLoS Pathogens, Vol. 20, Núm. 5
-
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy
Human gene therapy, Vol. 35, Núm. 17-18, pp. 582
2023
-
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
-
Cardiac Progenitor Cell Exosomal miR-935 Protects against Oxidative Stress
Cells, Vol. 12, Núm. 18
-
Exogenous aralar/slc25a12 can replace citrin/slc25a13 as malate aspartate shuttle component in liver
Molecular Genetics and Metabolism Reports, Vol. 35
-
Gene therapy for liver diseases — progress and challenges
Nature Reviews Gastroenterology and Hepatology, Vol. 20, Núm. 5, pp. 288-305
-
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
-
Photodynamic nasal SARS-CoV-2 decolonization shortens infectivity and influences specific T-Cell responses
Frontiers in Cellular and Infection Microbiology, Vol. 13
-
Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy
JHEP Reports, Vol. 5, Núm. 5
-
Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV)
Bone Marrow Transplantation
-
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
-
Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells
Pharmaceutics, Vol. 15, Núm. 10
-
Treatment with the senolytics dasatinib/quercetin reduces SARS-CoV-2-related mortality in mice
Aging Cell, Vol. 22, Núm. 3