Zobrazeno 1 - 10
of 207
pro vyhledávání: '"Alberto, Auricchio"'
Autor:
Eleonora Pedrazzoli, Michele Demozzi, Elisabetta Visentin, Matteo Ciciani, Ilaria Bonuzzi, Laura Pezzè, Lorenzo Lucchetta, Giulia Maule, Simone Amistadi, Federica Esposito, Mariangela Lupo, Annarita Miccio, Alberto Auricchio, Antonio Casini, Nicola Segata, Anna Cereseto
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-12 (2024)
Abstract The expansion of the CRISPR-Cas toolbox is highly needed to accelerate the development of therapies for genetic diseases. Here, through the interrogation of a massively expanded repository of metagenome-assembled genomes, mostly from human m
Externí odkaz:
https://doaj.org/article/b6397b430d9d4dcb8e5d106fca4eda64
Autor:
Rita Ferla, Fabio Dell’Aquila, Monica Doria, Maria Ferraiuolo, Alessia Noto, Fabiana Grazioli, Virginia Ammendola, Francesco Testa, Paolo Melillo, Carolina Iodice, Giulia Risca, Novella Tedesco, Pierre Romain le Brun, Enrico Maria Surace, Francesca Simonelli, Stefania Galimberti, Maria Grazia Valsecchi, Jean-Brice Marteau, Philippe Veron, Stefano Colloca, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 396-411 (2023)
Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large Myosin VIIA (MYO7A) gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which
Externí odkaz:
https://doaj.org/article/61581d1038c94d21b99808688a92c247
Autor:
Iolanda Boffa, Elena Polishchuk, Lucia De Stefano, Fabio Dell'Aquila, Edoardo Nusco, Elena Marrocco, Matteo Audano, Silvia Pedretti, Marianna Caterino, Ilaria Bellezza, Margherita Ruoppolo, Nico Mitro, Barbara Cellini, Alberto Auricchio, Nicola Brunetti‐Pierri
Publikováno v:
EMBO Molecular Medicine, Vol 15, Iss 4, Pp 1-11 (2023)
Abstract Gyrate atrophy of choroid and retina (GACR) is a chorioretinal degeneration caused by pathogenic variants in the gene encoding ornithine aminotransferase (OAT), an enzyme mainly expressed in liver. Affected patients have increased ornithine
Externí odkaz:
https://doaj.org/article/e07eec5ef9114bf685675cb9215d0081
Autor:
Sabrina Carrella, Martina Di Guida, Simona Brillante, Davide Piccolo, Ludovica Ciampi, Irene Guadagnino, Jorge Garcia Piqueras, Mariateresa Pizzo, Elena Marrocco, Marta Molinari, Georgios Petrogiannakis, Sara Barbato, Yulia Ezhova, Alberto Auricchio, Brunella Franco, Elvira De Leonibus, Enrico Maria Surace, Alessia Indrieri, Sandro Banfi
Publikováno v:
EMBO Molecular Medicine, Vol 14, Iss 11, Pp 1-19 (2022)
Abstract Inherited retinal diseases (IRDs) are a group of diseases whose common landmark is progressive photoreceptor loss. The development of gene‐specific therapies for IRDs is hampered by their wide genetic heterogeneity. Mitochondrial dysfuncti
Externí odkaz:
https://doaj.org/article/5ffcd1e1e6b74894ad6d8849c7cb89e8
Autor:
Agnese Padula, Raffaella Petruzzelli, Sasha A. Philbert, Stephanie J. Church, Federica Esposito, Severo Campione, Marcello Monti, Filomena Capolongo, Claudia Perna, Edoardo Nusco, Hartmut H. Schmidt, Alberto Auricchio, Garth J.S. Cooper, Roman Polishchuk, Pasquale Piccolo
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 495-504 (2022)
Wilson disease (WD) is a genetic disorder of copper homeostasis, caused by deficiency of the copper transporter ATP7B. Gene therapy with recombinant adeno-associated vectors (AAV) holds promises for WD treatment. However, the full-length human ATP7B
Externí odkaz:
https://doaj.org/article/9b06f42f269f4eb08bec78339d8b5af7
Autor:
Federica Esposito, Hristiana Lyubenova, Patrizia Tornabene, Stefano Auricchio, Antonella Iuliano, Edoardo Nusco, Simone Merlin, Cristina Olgasi, Giorgia Manni, Marco Gargaro, Francesca Fallarino, Antonia Follenzi, Alberto Auricchio
Publikováno v:
EMBO Molecular Medicine, Vol 14, Iss 6, Pp 1-15 (2022)
Abstract Liver gene therapy with adeno‐associated viral (AAV) vectors is under clinical investigation for haemophilia A (HemA), the most common inherited X‐linked bleeding disorder. Major limitations are the large size of the F8 transgene, which
Externí odkaz:
https://doaj.org/article/f569f4344bd64dee93d8db414a637859
Autor:
Patrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, Miriam Centrulo, Margherita Dell’Anno, Federica Esposito, Elena Marrocco, Emanuela Pone, Renato Minopoli, Carolina Iodice, Edoardo Nusco, Settimio Rossi, Hristiana Lyubenova, Anna Manfredi, Lucio Di Filippo, Antonella Iuliano, Annalaura Torella, Giulio Piluso, Francesco Musacchia, Enrico Maria Surace, Davide Cacchiarelli, Vincenzo Nigro, Alberto Auricchio
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
Limits of AAV-mediated gene therapy include targeting dominant mutations and inducing long-term transgene expression. Here, the authors show that AAV-HITI results in efficient allele-independent integration of a donor DNA in both retina and liver pro
Externí odkaz:
https://doaj.org/article/eff364ba6a29484688084b394c9c396c
Autor:
Patrizia Tornabene, Ivana Trapani, Miriam Centrulo, Elena Marrocco, Renato Minopoli, Mariangela Lupo, Carolina Iodice, Carlo Gesualdo, Francesca Simonelli, Enrico M. Surace, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 448-459 (2021)
Split intein-mediated protein trans-splicing expands AAV transfer capacity, thus overcoming the limited AAV cargo. However, non-mammalian inteins persist as trans-splicing by-products, and this could raise safety concerns for AAV intein clinical appl
Externí odkaz:
https://doaj.org/article/ff90e9e823594252bf35342e1bb5bf45
Autor:
Rita Ferla, Marialuisa Alliegro, Margherita Dell’Anno, Edoardo Nusco, John M. Cullen, Stephanie N. Smith, Tyra G. Wolfsberg, Patricia O’Donnell, Ping Wang, Anh-Dao Nguyen, Randy J. Chandler, Zelin Chen, Shawn M. Burgess, Charles H. Vite, Mark E. Haskins, Charles P. Venditti, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 247-257 (2021)
Adeno-associated viral (AAV) vectors have emerged as the preferred platform for in vivo gene transfer because of their combined efficacy and safety. However, insertional mutagenesis with the subsequent development of hepatocellular carcinomas (HCCs)
Externí odkaz:
https://doaj.org/article/bc88977cd5ef427da6506d192e84e042
Autor:
Francesca Gatto, Barbara Rossi, Antonietta Tarallo, Elena Polishchuk, Roman Polishchuk, Alessandra Carrella, Edoardo Nusco, Filomena Grazia Alvino, Francesca Iacobellis, Elvira De Leonibus, Alberto Auricchio, Graciana Diez-Roux, Andrea Ballabio, Giancarlo Parenti
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-12 (2017)
Abstract Pompe disease (PD) is a metabolic myopathy due to acid alpha-glucosidase deficiency and characterized by extensive glycogen storage and impaired autophagy. We previously showed that modulation of autophagy and lysosomal exocytosis by overexp
Externí odkaz:
https://doaj.org/article/678d39e7a19b43b9866916b4208c4792