S Cencig

Pall Life Sciences

Clarification with Pall Seitz® Depth Filters for Adherent and Suspension Adeno-Associated Virus Culture

V Nieto-Romero

Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria Fundación Jiménez. (IIS-FJD, UAM), 28040 Madrid, Spain

Restoration of glyoxylate detoxification capacity of induced-hepatocytes after gene editing of the AGXT gene: A potential treatment for Primary Hyperoxaluria type I

M I Garín

Division of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centre for Biomedical Network Research on Rare Diseases (CIBER-ER) and Advanced Therapy Unit, Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM).

Galectin-1 is required for the immune modulatory function of FOXP3+CD4+ Treg cells in the intestine

M I Garín

Division of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centre for Biomedical Network Research on Rare Diseases (CIBER-ER) and Advanced Therapy Unit, Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM).

Role of galectin-1 in regulatory T cell-based therapy in chronic colitis 

O Murillo

Center for Applied Medical Research

High value of [64Cu]CuCl₂ PET as a non-invasive tool to evaluate the restoration of physiological copper excretion after VTX-801 gene therapy in a mouse model of Wilson’s disease

L Neire

Vivet Therapeutics, S.L., Pamplona, Spain.

rAAV-mediated gene therapy in combination with short-term nitrogen-scavenger treatment corrects biochemical and behavioral abnormalities and increases lifespan in infant Citrullinemia Type 1 (CTLN-1) mice

M Hommel

Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, Pamplona, Spain; Institute for Sanitary Research (IdiSNA), Pamplona, Spain

Optimizing the IdeS treatment regimen for enhanced adeno-associated virus transduction in the presence of neutralizing antibodies

S Fuertes-Alvarez 

Biodonostia Institute

Terminal Schwann cells and Kranocytes connect neuromuscular junctions with the vascular network

E H de Laorden

Francisco de Vitoria University

Analysis of the neuroregenerative potential of human placental mesenchymal stem cells in a mammalian Central Nervous System model of axotomy

L Blazquez

Biodonostia Institute

Investigation of RNA-misprocessing in neurological diseases for the development of novel diagnostic and therapeutic approaches

A Velasco Bilbao

Biodonostia Institute

Molecular characterization of a non-coding mutation in GRN gene and investigation of CRISPR-dCas13 system for splicing modulation

N Pelaez-Poblet 

Biodonostia Institute

Characterizing novel markers for Kranocytes, the "forgotten" NMJ-capping cells