Nanoparticle Delivery of Antisense Oligonucleotides and Their Application in the Exon Skipping Strategy for Duchenne Muscular Dystrophy

Por Falzarano, Maria Sofia, Passarelli, Chiara, Ferlini, Alessandra
Publicado em 2014

Biodistribution Studies of Polymeric Nanoparticles for Drug Delivery in Mice

Por Falzarano, Maria Sofia, Bassi, Elena, Passarelli, Chiara, Braghetta, Paola, Ferlini, Alessandra
Publicado no Hum Gene Ther (2014)

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 em 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 em 2013

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 em 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 em 2012

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 em 2014

Deep RNA profiling identified CLOCK and molecular clock genes as pathophysiological signatures in collagen VI myopathy

Por Scotton, Chiara, Bovolenta, Matteo, Schwartz, Elena, Falzarano, Maria Sofia, Martoni, Elena, Passarelli, Chiara, Armaroli, Annarita, Osman, Hana, Rodolico, Carmelo, Messina, Sonia, Pegoraro, Elena, D'Amico, Adele, Bertini, Enrico, Gualandi, Francesca, Neri, Marcella, Selvatici, Rita, Boffi, Patrizia, Maioli, Maria Antonietta, Lochmüller, Hanns, Straub, Volker, Bushby, Katherine, Castrignanò, Tiziana, Pesole, Graziano, Sabatelli, Patrizia, Merlini, Luciano, Braghetta, Paola, Bonaldo, Paolo, Bernardi, Paolo, Foley, Reghan, Cirak, Sebahattin, Zaharieva, Irina, Muntoni, Francesco, Capitanio, Daniele, Gelfi, Cecilia, Kotelnikova, Ekaterina, Yuryev, Anton, Lebowitz, Michael, Zhang, Xiping, Hodge, Brian A., Esser, Karyn A., Ferlini, Alessandra
Publicado no J Cell Sci (2016)