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Human Gene Therapy Methods|August 17, 2013
Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn ratsNunzia Pastore, Edoardo Nusco, Pasquale Piccolo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2024
Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retinaFabio Dell'Aquila, Roberto Di Cunto, Elena Marrocco, et al.
Proceedings of the National Academy of Sciences of the United States of America|March 16, 2007
Systemic inflammation and neurodegeneration in a mouse model of multiple sulfatase deficiencyCarmine Settembre, Ida Annunziata, Carmine Spampanato, et al.
Science Translational Medicine|March 8, 2013
Phenylbutyrate therapy for pyruvate dehydrogenase complex deficiency and lactic acidosisRosa Ferriero, Giuseppe Manco, Eleonora Lamantea, et al.
The Biochemical Journal|January 9, 2007
SUMF1 enhances sulfatase activities in vivo in five sulfatase deficienciesAlessandro Fraldi, Alessandra Biffi, Alessia Lombardi, et al.
Molecular Therapy. Methods & Clinical Development|September 22, 2017
Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VIRita Ferla, Marialuisa Alliegro, Jean-Brice Marteau, et al.
The Journal of Biological Chemistry|July 30, 2020
CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicitySergio Attanasio, Rosa Ferriero, Gwladys Gernoux, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 18, 2011
Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorderCarmine Spampanato, Elvira De Leonibus, Paola Dama, et al.
EMBO Molecular Medicine|April 10, 2013
A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIANicolina Cristina Sorrentino, Luca D'Orsi, Irene Sambri, et al.
EMBO Molecular Medicine|May 2, 2022
Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia AFederica Esposito, Hristiana Lyubenova, Patrizia Tornabene, et al.
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