Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Virginia Haurigot, Sara Marcó, Albert Ribera, +16

Journal of Clinical Investigation

ImmunologyExperimental PathologyPathologyTranslational MedicineMps Iiia +14

Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer

Albert Ruzo, Sara Marcó, Miquel García, +7

Human Gene Therapy

Aav9 Gene TransferImmunologyPathologyPeripheral NervesMucopolysaccharidosis Type Iiia +15

Long-Term Retinal PEDF Overexpression Prevents Neovascularization in a Murine Adult Model of Retinopathy

Virginia Haurigot, Pilar Villacampa, Albert Ribera, +4

PLoS ONE

Diabetic RetinopathyOcular DiseaseRetinaOphthalmologyExperimental Ophthalmology +13

Angiocrine polyamine production regulates adiposity

Erika Monelli, Pilar Villacampa, Amaia Zabala‐Letona, +27

Nature Metabolism

ObesityMetabolic SyndromeAngiocrine Polyamine ProductionMolecular PhysiologyPhysiology +5

Phosphoinositide 3-Kinase–Regulated Pericyte Maturation Governs Vascular Remodeling

Ana M. Figueiredo, Pilar Villacampa, Rodrigo Diéguez‐Hurtado, +11

Circulation

Increased Intraocular Insulin-like Growth Factor-I Triggers Blood-Retinal Barrier Breakdown

Virginia Haurigot, Pilar Villacampa, Albert Ribera, +9

Journal of Biological Chemistry

Myeloid-Derived Vascular Endothelial Growth Factor and Hypoxia-Inducible Factor Are Dispensable for Ocular Neovascularization—Brief Report

Sidath E. Liyanage, Alessandro Fantin, Pilar Villacampa, +8

Arteriosclerosis Thrombosis and Vascular Biology

Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy

Albert Ribera, Virginia Haurigot, Miquel García, +15

Human Molecular Genetics

NeurogenomicsImmunologyPathologyNeurochemical BiomarkersSynaptic Signaling +24

Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice

Albert Ruzo, Miquel García, Albert Ribera, +10

Molecular Therapy

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID

Carles Roca, Sandra Motas, Sara Marcó, +12

Human Molecular Genetics

In Vivo Gene TherapyHuman MpsiiidImmunodeficienciesGeneticsImmunology +18