Urinary Stem Cells as Tools to Study Genetic Disease: Overview of the Literature

por Falzarano, Maria Sofia, Ferlini, Alessandra
Publicado 2019

Duchenne Muscular Dystrophy: From Diagnosis to Therapy

por Falzarano, Maria Sofia, Scotton, Chiara, Passarelli, Chiara, Ferlini, Alessandra
Publicado 2015

Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy

por Fortunato, Fernanda, Rossi, Rachele, Falzarano, Maria Sofia, Ferlini, Alessandra
Publicado 2021

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

por Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Publicado 2017

Tracers for Cardiac Imaging: Targeting the Future of Viable Myocardium

por Nappi, Carmela, Panico, Mariarosaria, Falzarano, Maria, Vallone, Carlo, Ponsiglione, Andrea, Cutillo, Paolo, Zampella, Emilia, Petretta, Mario, Cuocolo, Alberto
Publicado 2023

Intramedullary Spinal Cord Metastases from Differentiated Thyroid Cancer, a Case Report

por Volpe, Fabio, Piscopo, Leandra, Manganelli, Mariarosaria, Falzarano, Maria, Volpicelli, Federica, Nappi, Carmela, Imbriaco, Massimo, Cuocolo, Alberto, Klain, Michele
Publicado 2022

mRNA in situ hybridization exhibits unbalanced nuclear/cytoplasmic dystrophin transcript repartition in Duchenne myogenic cells and skeletal muscle biopsies

por Falzarano, Maria Sofia, Mietto, Martina, Fortunato, Fernanda, Farnè, Marianna, Martini, Fernanda, Ala, Pierpaolo, Selvatici, Rita, Muntoni, Francesco, Ferlini, Alessandra
Publicado 2023

Efficacy of Empirical Radioiodine Therapy in Patients with Differentiated Thyroid Cancer and Elevated Serum Thyroglobulin without Evidence of Structural Disease: A Propensity Score...

por Piscopo, Leandra, Zampella, Emilia, Volpe, Fabio, Gaudieri, Valeria, Nappi, Carmela, Cutillo, Paolo, Volpicelli, Federica, Falzarano, Maria, Pace, Leonardo, Cuocolo, Alberto, Klain, Michele
Publicado 2023

The DMD Locus Harbours Multiple Long Non-Coding RNAs Which Orchestrate and Control Transcription of Muscle Dystrophin mRNA Isoforms

por Bovolenta, Matteo, Erriquez, Daniela, Valli, Emanuele, Brioschi, Simona, Scotton, Chiara, Neri, Marcella, Falzarano, Maria Sofia, Gherardi, Samuele, Fabris, Marina, Rimessi, Paola, Gualandi, Francesca, Perini, Giovanni, Ferlini, Alessandra
Publicado 2012

Biodistribution and Molecular Studies on Orally Administered Nanoparticle-AON Complexes Encapsulated with Alginate Aiming at Inducing Dystrophin Rescue in mdx Mice

por Falzarano, Maria Sofia, Passarelli, Chiara, Bassi, Elena, Fabris, Marina, Perrone, Daniela, Sabatelli, Patrizia, Maraldi, Nadir M., Donà, Silvia, Selvatici, Rita, Bonaldo, Paolo, Sparnacci, Katia, Laus, Michele, Braghetta, Paola, Rimessi, Paola, Ferlini, Alessandra
Publicado 2013

Urine-Derived Stem Cells Express 571 Neuromuscular Disorders Causing Genes, Making Them a Potential in vitro Model for Rare Genetic Diseases

por Falzarano, Maria Sofia, Rossi, Rachele, Grilli, Andrea, Fang, Mingyan, Osman, Hana, Sabatelli, Patrizia, Antoniel, Manuela, Lu, Zhiyuan, Li, Wenyan, Selvatici, Rita, Al-Khalili, Cristina, Gualandi, Francesca, Bicciato, Silvio, Torelli, Silvia, Ferlini, Alessandra
Publicado 2021

Circadian Genes as Exploratory Biomarkers in DMD: Results From Both the mdx Mouse Model and Patients

por Rossi, Rachele, Falzarano, Maria Sofia, Osman, Hana, Armaroli, Annarita, Scotton, Chiara, Mantuano, Paola, Boccanegra, Brigida, Cappellari, Ornella, Schwartz, Elena, Yuryev, Anton, Mercuri, Eugenio, Bertini, Enrico, D’Amico, Adele, Mora, Marina, Johansson, Camilla, Al-Khalili Szigyarto, Cristina, De Luca, Annamaria, Ferlini, Alessandra
Publicado 2021

A novel custom high density-comparative genomic hybridization array detects common rearrangements as well as deep intronic mutations in dystrophinopathies

por Bovolenta, Matteo, Neri, Marcella, Fini, Sergio, Fabris, Marina, Trabanelli, Cecilia, Venturoli, Anna, Martoni, Elena, Bassi, Elena, Spitali, Pietro, Brioschi, Simona, Falzarano, Maria S, Rimessi, Paola, Ciccone, Roberto, Ashton, Emma, McCauley, Joanne, Yau, Shu, Abbs, Stephen, Muntoni, Francesco, Merlini, Luciano, Gualandi, Francesca, Ferlini, Alessandra
Publicado 2008

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype

por Brioschi, Simona, Gualandi, Francesca, Scotton, Chiara, Armaroli, Annarita, Bovolenta, Matteo, Falzarano, Maria S, Sabatelli, Patrizia, Selvatici, Rita, D’Amico, Adele, Pane, Marika, Ricci, Giulia, Siciliano, Gabriele, Tedeschi, Silvana, Pini, Antonella, Vercelli, Liliana, De Grandis, Domenico, Mercuri, Eugenio, Bertini, Enrico, Merlini, Luciano, Mongini, Tiziana, Ferlini, Alessandra
Publicado 2012

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures

por Hiller, Monika, Falzarano, Maria Sofia, Garcia-Jimenez, Iker, Sardone, Valentina, Verheul, Ruurd C., Popplewell, Linda, Anthony, Karen, Ruiz-Del-Yerro, Estibaliz, Osman, Hana, Goeman, Jelle J., Mamchaoui, Kamel, Dickson, George, Ferlini, Alessandra, Muntoni, Francesco, Aartsma-Rus, Annemieke, Arechavala-Gomeza, Virginia, Datson, Nicole A., Spitali, Pietro
Publicado 2018

Homozygous Recessive Versican Missense Variation Is Associated With Early Teeth Loss in a Pakistani Family

por Bigoni, Stefania, Neri, Marcella, Scotton, Chiara, Farina, Roberto, Sabatelli, Patrizia, Jiang, Chongyi, Zhang, Jianguo, Falzarano, Maria Sofia, Rossi, Rachele, Ognibene, Davide, Selvatici, Rita, Gualandi, Francesca, Bosshardt, Dieter, Perri, Paolo, Campa, Claudio, Brancati, Francesco, Salvatore, Marco, De Stefano, Maria Chiara, Taruscio, Domenica, Trombelli, Leonardo, Fang, Mingyan, Ferlini, Alessandra
Publicado 2019

RNA-seq in DMD urinary stem cells recognized muscle-related transcription signatures and addressed the identification of atypical mutations by whole-genome sequencing

por Falzarano, Maria S., Grilli, Andrea, Zia, Silvia, Fang, Mingyan, Rossi, Rachele, Gualandi, Francesca, Rimessi, Paola, El Dani, Reem, Fabris, Marina, Lu, Zhiyuan, Li, Wenyan, Mongini, Tiziana, Ricci, Federica, Pegoraro, Elena, Bello, Luca, Barp, Andrea, Sansone, Valeria A., Hegde, Madhuri, Roda, Barbara, Reschiglian, Pierluigi, Bicciato, Silvio, Selvatici, Rita, Ferlini, Alessandra
Publicado 2021

A novel DMD IRES results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations

por Wein, Nicolas, Vulin, Adeline, Sofia Falzarano, Maria, Al-Khalili Szigyarto, Christina, Maiti, Baijayanta, Findlay, Andrew, Heller, Kristin N, Uhlén, Mathias, Bakthavachalu, Baskar, Messina, Sonia, Vita, Giuseppe, Passarelli, Chiara, Gualandi, Francesca, Wilton, Steve D, Rodino-Klapac, Louise, Yang, Lin, Dunn, Diane M., Schoenberg, Daniel, Weiss, Robert B., Howard, Michael T., Ferlini, Alessandra, Flanigan, Kevin M.
Publicado 2014

Dystrophin involvement in peripheral circadian SRF signalling

por Betts, Corinne A, Jagannath, Aarti, van Westering, Tirsa LE, Bowerman, Melissa, Banerjee, Subhashis, Meng, Jinhong, Falzarano, Maria Sofia, Cravo, Lara, McClorey, Graham, Meijboom, Katharina E, Bhomra, Amarjit, Lim, Wooi Fang, Rinaldi, Carlo, Counsell, John R, Chwalenia, Katarzyna, O’Donovan, Elizabeth, Saleh, Amer F, Gait, Michael J, Morgan, Jennifer E, Ferlini, Alessandra, Foster, Russell G, Wood, Matthew JA
Publicado 2021

Tumor Necrosis Factor Receptor SF10A (TNFRSF10A) SNPs Correlate With Corticosteroid Response in Duchenne Muscular Dystrophy

por Passarelli, Chiara, Selvatici, Rita, Carrieri, Alberto, Di Raimo, Francesca Romana, Falzarano, Maria Sofia, Fortunato, Fernanda, Rossi, Rachele, Straub, Volker, Bushby, Katie, Reza, Mojgan, Zharaieva, Irina, D’Amico, Adele, Bertini, Enrico, Merlini, Luciano, Sabatelli, Patrizia, Borgiani, Paola, Novelli, Giuseppe, Messina, Sonia, Pane, Marika, Mercuri, Eugenio, Claustres, Mireille, Tuffery-Giraud, Sylvie, Aartsma-Rus, Annemieke, Spitali, Pietro, T’Hoen, Peter A. C., Lochmüller, Hanns, Strandberg, Kristin, Al-Khalili, Cristina, Kotelnikova, Ekaterina, Lebowitz, Michael, Schwartz, Elena, Muntoni, Francesco, Scapoli, Chiara, Ferlini, Alessandra
Publicado 2020