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Science Advances|January 1, 2026
TFEB coordinates autophagosome biogenesis and ribophagy during starvation via SQSTM1Maria Iavazzo, Laura Cinque, Sophie Levantovsky, et al.
Molecular Therapy. Methods & Clinical Development|September 12, 2022
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in miceAgnese Padula, Raffaella Petruzzelli, Sasha A Philbert, et al.
Molecular Therapy. Methods & Clinical Development|December 3, 2019
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIANicolina Cristina Sorrentino, Vincenzo Cacace, Maria De Risi, et al.
Elife|September 27, 2016
Modelling TFE renal cell carcinoma in mice reveals a critical role of WNT signalingAlessia Calcagnì, Lotte Kors, Eric Verschuren, et al.
Journal of the American Society of Nephrology : JASN|April 1, 2025
TRPML-1 Dysfunction and Renal Tubulopathy in Mucolipidosis Type IVGiuseppina Grieco, Sandro Montefusco, Edoardo Nusco, et al.
Molecular Therapy. Methods & Clinical Development|January 21, 2021
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectorsRita Ferla, Marialuisa Alliegro, Margherita Dell'Anno, 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 the American Society of Nephrology : JASN|December 4, 2024
TRPML-1 Dysfunction and Renal Tubulopathy in Mucolipidosis Type IVGiuseppina Grieco, Sandro Montefusco, Edoardo Nusco, et al.
Nature|July 3, 2020
A substrate-specific mTORC1 pathway underlies Birt-Hogg-Dubé syndromeGennaro Napolitano, Chiara Di Malta, Alessandra Esposito, et al.
The EMBO Journal|March 11, 2020
Light-responsive microRNA miR-211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearanceFederica Naso, Daniela Intartaglia, Danila Falanga, et al.
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