Publications (72) Publications in which a researcher has participated

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2025

  1. Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group

    Molecular Therapy Nucleic Acids, Vol. 36, Núm. 1

2024

  1. 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

  2. Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1

    EMBO molecular medicine, Vol. 16, Núm. 1, pp. 112-131

  3. 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

  4. GCN2 inhibition reduces mutant SOD1 clustering and toxicity and delays disease progression in an amyotrophic lateral sclerosis mouse model

    Translational Neurodegeneration

  5. Gene Therapy

    Topics in Medicinal Chemistry (Springer Science and Business Media Deutschland GmbH), pp. 187-211

  6. Gene editing in liver diseases

    FEBS Letters, Vol. 598, Núm. 19, pp. 2348-2371

  7. Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

    Nature Communications, Vol. 15, Núm. 1

  8. In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells

    International Journal of Molecular Sciences, Vol. 25, Núm. 23

  9. Profile of plasma microRNAs as a potential biomarker of Wilson’s disease

    Journal of Gastroenterology, Vol. 59, Núm. 10, pp. 921-931

  10. Protective role of RIPK1 scaffolding against HDV-induced hepatocyte cell death and the significance of cytokines in mice

    PLoS Pathogens, Vol. 20, Núm. 5

  11. Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy

    Human gene therapy, Vol. 35, Núm. 17-18, pp. 582

2023

  1. AG5 is a potent non-steroidal anti-inflammatory and immune regulator that preserves innate immunity

    Biomedicine and Pharmacotherapy, Vol. 169

  2. 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

  3. Cardiac Progenitor Cell Exosomal miR-935 Protects against Oxidative Stress

    Cells, Vol. 12, Núm. 18

  4. Exogenous aralar/slc25a12 can replace citrin/slc25a13 as malate aspartate shuttle component in liver

    Molecular Genetics and Metabolism Reports, Vol. 35

  5. Gene therapy for liver diseases — progress and challenges

    Nature Reviews Gastroenterology and Hepatology, Vol. 20, Núm. 5, pp. 288-305

  6. 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

  7. Photodynamic nasal SARS-CoV-2 decolonization shortens infectivity and influences specific T-Cell responses

    Frontiers in Cellular and Infection Microbiology, Vol. 13

  8. Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy

    JHEP Reports, Vol. 5, Núm. 5