Sfoglia per Autore
Preclinical Development of an Ex Vivo Gene Therapy for Mucopolysaccharidosis Type II
2022 Das, S; Rruga, F; Rigon, L; Patel, J; Milazzo, R; Poletti, V; Biffi, A
Drosophila d-idua reduction mimics mucopolysaccharidosis type i disease-related phenotypes
2022 De Filippis, C.; Napoli, B.; Rigon, L.; Guarato, G.; Bauer, R.; Tomanin, R.; Orso, G.
Development of an Ex Vivo Gene Therapy for Frontotemporal Dementia (FTD)
2022 Ciervo, Y; Rigon, L; Spadini, S; Accardo, M; Milazzo, R; Biffi, A
Hematopoietic Stem and Progenitor Cell Gene Therapy Uniquely Benefits Multiple Sclerosis in the Animal Model
2022 Spadini, S; Milazzo, R; Rigon, L; Accardo, M; Santinon, G; Ciervo, Y; Costa, A; Peviani, M; Ben Nasr, M; Rizzardi, Gp; Fiorina, P; Biffi, A
Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy
2022 Maccari, F.; Rigon, L.; Mantovani, V.; Galeotti, F.; Salvalaio, M.; D'Avanzo, F.; Zanetti, A.; Capitani, F.; Gabrielli, O.; Tomanin, R.; Volpi, N.
Exploiting the potential of drosophila models in lysosomal storage disorders: Pathological mechanisms and drug discovery
2021 Rigon, L.; De Filippis, C.; Napoli, B.; Tomanin, R.; Orso, G.
Modeling Mucopolysaccharidosis Type II in the Fruit Fly by Using the RNA Interference Approach
2020 Rigon, Laura; Kucharowski, Nicole; Eckardt, Franka; Bauer, Reinhard
Mucopolysaccharidosis type II: One hundred years of research, diagnosis, and treatment
2020 Dʹavanzo, F.; Rigon, L.; Zanetti, A.; Tomanin, R.
Molecular diagnosis of patients affected by mucopolysaccharidosis: a multicenter study
2019 Zanetti, A.; D'Avanzo, F.; Rigon, L.; Rampazzo, A.; Concolino, D.; Barone, R.; Volpi, N.; Santoro, L.; Lualdi, S.; Bertola, F.; Scarpa, M.; Tomanin, R.
Targeting brain disease in MPSII: Preclinical evaluation of IDS-loaded PLGA nanoparticles
2019 Rigon, Laura; Salvalaio, Marika; Pederzoli, Francesca; Legnini, Elisa; Duskey, Jason Thomas; D'Avanzo, Francesca; De Filippis, Concetta; Ruozi, Barbara; Marin, Oriano; Vandelli, Maria Angela; Ottonelli, Ilaria; Scarpa, Maurizio; Tosi, Giovanni; Tomanin, Rosella
FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII).
2018 Bellesso, Stefania; Salvalaio, Marika; Lualdi, Susanaa; Tognon, Elisa; Costa, Roberto; Braghetta, Paola; Giraudo, Chiara; Stramare, Roberto; Rigon, Laura; Filocamo, Mirella; Tomanin, Rosella; Moro, Enrico
Composition and structure of glycosaminoglycans in DBS from 2-3-day-old newborns for the diagnosis of mucopolysaccharidosis
2018 Maccari, F.; Galeotti, F.; Mantovani, V.; Zampini, L.; Padella, L.; Rigon, L.; Concolino, D.; Fiumara, A.; Pascale, E.; Pittala, A.; Galeazzi, T.; Monachesi, C.; Marchesiello, R. L.; Coppa, G.; Gabrielli, O.; Volpi, N.
Corrigendum: FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII) [Human Molecular Genetics, 27, 13, (2018) (2262-2275)] DOI: 10.1093/hmg/ddy131
2018 Bellesso, S.; Salvalaio, M.; Lualdi, S.; Tognon, E.; Costa, R.; Braghetta, P.; Giraudo, C.; Stramare, R.; Rigon, L.; Filocamo, M.; Tomanin, R.; Moro, E.
Brain RNA-seq profiling of the mucopolysaccharidosis type II mouse model
2017 Salvalaio, Marika; D’Avanzo, Francesca; Rigon, Laura; Zanetti, Alessandra; D’Angelo, Michela; Valle, Giorgio; Scarpa, Maurizio; Tomanin, Rosella
Early diagnosis of mucopolysaccharidoses in developing countries: A low cost and easy execution approach
2017 Gabrielli, O.; Zampini, L.; Monachesi, C.; Marchesiello, R. L.; Padella, L.; Santoro, L.; Volpi, N.; Concolino, D.; Fiumara, A.; Rigon, L.; Mazzoli, M.; Carnielli, V. P.; Giovagnoni, A.; Catassi, C.; Galeazzi, T.; Coppa, G. V.
Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders
2016 Salvalaio, M; Rigon, Laura; Belletti, D; D'Avanzo, Francesca; Pederzoli, F; Ruozi, B; Marin, Oriano; Vandelli, Ma; Forni, F; Scarpa, Maurizio; Tomanin, Rosella; Tosi, G.
Nanotechnologies for CNS drug delivery: therapy for the neurological compartment in the Mucopolisaccharidoses
2015 Rigon, Laura
BBB Crossing in Lysosomal Storage Disorders: A Nanoparticle-Based Approach
2014 Rigon, Laura; Salvalaio, Marika; Tosi, Giovanni; Belletti, Daniela; D'Avanzo, Francesca; Ruozi, Barbara; Vandelli, Maria Angela; Forni, Flavio; Scarpa, Maurizio; Tomanin, Rosella
Circadian transcriptome analysis in human fibroblasts from Hunter syndrome and impact of iduronate-2-sulfatase treatment
2013 Mazzoccoli, G; Tomanin, Rosella; Mazza, T; D'Avanzo, Francesca; Salvalaio, Marika; Rigon, Laura; Zanetti, Alessandra; Pazienza, V; Francavilla, M; Giuliani, F; Vinciguerra, M; Scarpa, Maurizio
LGI1 microdeletion in autosomal dominant lateral temporal epilepsy.
2012 Fanciulli, M; Santulli, L; Errichiello, L; Barozzi, C; Tomasi, L; Rigon, Laura; Cubeddu, T; de Falco, A; Rampazzo, Alessandra; Michelucci, R; Uzzau, S; Striano, S; de Falco, Fa; Striano, P; Nobile, C.
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