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Scientific Reports|November 10, 2017
AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe DiseaseFrancesca Gatto, Barbara Rossi, Antonietta Tarallo, et al.Scientific Reports|April 3, 2026
Crosstalk between inflammation and autophagy in CeD organoidsClaudia Bellomo, Francesca Furone, Renata Auricchio, et al.Acta Neuropathologica Communications|November 9, 2018
Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe diseaseGerben J Schaaf, Tom J M van Gestel, Stijn L M In 't Groen, et al.Proceedings of the National Academy of Sciences of the United States of America|April 15, 2026
Uncovering mitochondrial defects in photoreceptors opens therapeutic opportunities for Stargardt diseaseSimona Brillante, Mariagrazia Volpe, Anna Diana, et al.Frontiers in Immunology|February 7, 2025
Characterization of a WAS splice-site variant in a patient with Wiskott-Aldrich syndromeElisabetta Toriello, Rosa Maritato, Antonio De Rosa, et al.EMBO Molecular Medicine|October 4, 2021
Correction of oxidative stress enhances enzyme replacement therapy in Pompe diseaseAntonietta Tarallo, Carla Damiano, Sandra Strollo, et al.Journal of Medicinal Chemistry|September 15, 2025
C-Branched Iminosugars as Selective Pharmacological Chaperones of Lysosomal α-Glucosidase for the Treatment of Pompe DiseaseAnaïs Vieira Da Cruz, Valentine Perraudin, Nadia Minopoli, et al.Plos One|May 20, 2020
Aortopathies in mouse models of Pompe, Fabry and Mucopolysaccharidosis IIIB lysosomal storage diseasesMaria Paola Belfiore, Francesca Iacobellis, Emma Acampora, et al.Developmental Cell|June 10, 2014
Wilson disease protein ATP7B utilizes lysosomal exocytosis to maintain copper homeostasisElena V Polishchuk, Mafalda Concilli, Simona Iacobacci, et al.The Journal of Allergy and Clinical Immunology|December 2, 2023
How dietary advanced glycation end products could facilitate the occurrence of food allergyLorella Paparo, Serena Coppola, Rita Nocerino, et al.Pageof 3