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Biochimica Et Biophysica Acta. Molecular Basis of Disease|June 27, 2025
Normal urinary oxalate excretion in 4-hydroxy-2-oxo-glutarate aldolase 1 (HOGA1) deficient mice with AGT expression in peroxisomes and not in mitochondriaIolanda Boffa, Rosa Ferriero, Mariarosaria Cancelliere, et al.
Molecular Therapy. Nucleic Acids|July 10, 2025
Hepatocyte delivery of miR-34b/c reduces hepatic stellate cell activation and improves liver fibrosisPasquale Piccolo, Rosa Ferriero, Claudia Perna, et al.
Proceedings of the National Academy of Sciences of the United States of America|December 28, 2017
Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemiaLeandro R Soria, Gabriella Allegri, Dominique Melck, et al.
JCI Insight|September 14, 2023
Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson diseaseAgnese Padula, Michele Spinelli, Edoardo Nusco, et al.
Nature|November 24, 2015
FGF signalling regulates bone growth through autophagyLaura Cinque, Alison Forrester, Rosa Bartolomeo, et al.
The Journal of Clinical Investigation|September 6, 2017
mTORC1 hyperactivation arrests bone growth in lysosomal storage disorders by suppressing autophagyRosa Bartolomeo, Laura Cinque, Chiara De Leonibus, et al.
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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 23, 2020
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage DiseaseAntonio Monaco, Veronica Maffia, Nicolina Cristina Sorrentino, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 3, 2023
Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsinNunzia Pastore, Francesco Annunziata, Rita Colonna, et al.
EMBO Molecular Medicine|January 17, 2023
Liver-directed gene therapy for ornithine aminotransferase deficiencyIolanda Boffa, Elena Polishchuk, Lucia De Stefano, et al.
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