Publicaciones en colaboración con investigadores/as de Centro de Investigación Médica Aplicada (112)

2024

  1. Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles

    Gene Therapy, Vol. 31, Núm. 9-10, pp. 455-466

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

    Translational Neurodegeneration

  3. Pharmacological inhibition of the integrated stress response accelerates disease progression in an amyotrophic lateral sclerosis mouse model

    British Journal of Pharmacology, Vol. 181, Núm. 3, pp. 495-508

  4. Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates

    Gut

2023

  1. Engineering U1-Based Tetracycline-Inducible Riboswitches to Control Gene Expression in Mammals

    ACS Nano, Vol. 17, Núm. 23, pp. 23331-23346

  2. Glutamatergic and GABAergic Receptor Modulation Present Unique Electrophysiological Fingerprints in a Concentration-Dependent and Region-Specific Manner

    eNeuro, Vol. 10, Núm. 4

2022

  1. Galectin-3 inhibition boosts the therapeutic efficacy of Semliki Forest virus in pediatric osteosarcoma

    Molecular Therapy - Oncolytics, Vol. 26, pp. 246-264

  2. 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

  3. Oncolytic DNX-2401 Virus for Pediatric Diffuse Intrinsic Pontine Glioma

    New England Journal of Medicine, Vol. 386, Núm. 26, pp. 2471-2481

2021

  1. Imaging of Stroke in Rodents Using a Clinical Scanner and Inductively Coupled Specially Designed Receiver Coils

    Annals of Biomedical Engineering, Vol. 49, Núm. 2, pp. 746-756

  2. Local administration of IL-12 with an HC vector results in local and metastatic tumor control in pediatric osteosarcoma

    Molecular Therapy - Oncolytics, Vol. 20, pp. 23-33

  3. Neuroplasticity during the transition period: How the adolescent brain can recover from aphasia. A pilot study

    Brain and Development, Vol. 43, Núm. 4, pp. 556-562

  4. Transfer of SCN1A to the brain of adolescent mouse model of Dravet syndrome improves epileptic, motor, and behavioral manifestations

    Molecular Therapy - Nucleic Acids, Vol. 25, pp. 585-602

  5. Understanding the molecular mechanism of mir-877-3p could provide potential biomarkers and therapeutic targets in squamous cell carcinoma of the cervix

    Cancers, Vol. 13, Núm. 7

2020

  1. Abnormal brain gamma oscillations in response to auditory stimulation in Dravet syndrome

    European Journal of Paediatric Neurology, Vol. 24, pp. 134-141

  2. Alterations of the Hippocampal Neurogenic Niche in a Mouse Model of Dravet Syndrome

    Frontiers in Cell and Developmental Biology, Vol. 8

  3. Cholinergic midbrain afferents modulate striatal circuits and shape encoding of action strategies

    Nature Communications, Vol. 11, Núm. 1

2019

  1. Danio rerio as model organism for adenoviral vector evaluation

    Genes, Vol. 10, Núm. 12

  2. 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

2018

  1. An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria

    Human Gene Therapy, Vol. 29, Núm. 4, pp. 480-491