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Molecular Therapy : the Journal of the American Society of Gene Therapy|September 24, 2016
Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage DiseaseMarialuisa Alliegro, Rita Ferla, Edoardo Nusco, et al.Human Molecular Genetics|August 16, 2013
Tbx1 regulates brain vascularizationSara Cioffi, Stefania Martucciello, Filomena Gabriella Fulcoli, et al.Journal of Hepatology|March 28, 2018
Pyruvate dehydrogenase complex and lactate dehydrogenase are targets for therapy of acute liver failureRosa Ferriero, Edoardo Nusco, Rossella De Cegli, et al.Human Molecular Genetics|January 5, 2012
Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseasesRaquel de Pablo-Latorre, Assunta Saide, Elena V Polishhuck, et al.Human Molecular Genetics|September 30, 2010
Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapyVinicia Assunta Polito, Serena Abbondante, Roman S Polishchuk, et al.Frontiers in Aging Neuroscience|July 18, 2022
Induction of Autophagy Promotes Clearance of RHOP23H Aggregates and Protects From Retinal DegenerationDaniela Intartaglia, Giuliana Giamundo, Federica Naso, et al.Frontiers in Cell and Developmental Biology|March 21, 2020
Retinal Degeneration in MPS-IIIA Mouse ModelDaniela Intartaglia, Giuliana Giamundo, Elena Marrocco, et al.American Journal of Medical Genetics. Part A|May 19, 2009
Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIBCarmela Di Domenico, Guglielmo R D Villani, Daniele Di Napoli, et al.Human Gene Therapy|July 7, 2012
Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscleNunzia Pastore, Edoardo Nusco, Jana Vaníkova, et al.Plos One|April 1, 2009
Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatmentsCarmen Vitiello, Stefania Faraso, Nicolina Cristina Sorrentino, et al.Pageof 5