Zobrazeno 1 - 10
of 27
pro vyhledávání: '"Rita Ferla"'
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:
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:
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:
Rita Ferla, Marialuisa Alliegro, Jean-Brice Marteau, Margherita Dell’Anno, Edoardo Nusco, Severine Pouillot, Stefania Galimberti, Maria Grazia Valsecchi, Vincent Zuliani, Alberto Auricchio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 143-158 (2017)
In vivo gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. We recently demonstrated that AAV8-mediated liver gene transfer is effective in animal models of mucopolysaccharidosis type VI (MPS VI), a rare lysosomal
Externí odkaz:
https://doaj.org/article/2e1487119ed54b2f941a430432729950
Autor:
Nicola Brunetti-Pierri, Rita Ferla, Virginia Maria Ginocchio, Alessandro Rossi, Simona Fecarotta, Roberta Romano, Giancarlo Parenti, Yilmaz Yildiz, Stefano Zancan, Valentina Pecorella, Margherita Dell’Anno, Mafalda Graziano, Marialuisa Alliegro, Generoso Andria, Francesca Santamaria, Raffaella Brunetti-Pierri, Francesca Simonelli, Vincenzo Nigro, Maria Vargas, Giuseppe Servillo, Francesco Borgia, Ernesto Soscia, Marco Gargaro, Silvia Funghini, Novella Tedesco, Pierre Romain Le Brun, Charles A. Rupar, Chitra Prasad, Mar O’Callaghan, John J. Mitchell, Olivier Danos, Jean-Brice Marteau, Stefania Galimberti, Maria Grazia Valsecchi, Philippe Veron, Federico Mingozzi, Francesca Fallarino, Giancarlo la Marca, H. Serap Sivri, Alberto Auricchio
Publikováno v:
NEJM Evidence. 1
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::93d416c60dd7074db1fccb900afbb5ac
https://doi.org/10.1056/evidoa2200052
https://doi.org/10.1056/evidoa2200052
Autor:
Zelin Chen, Charles P. Venditti, John M. Cullen, Ping Wang, Stephanie Smith, Rita Ferla, Tyra G. Wolfsberg, Anh-Dao Nguyen, Alberto Auricchio, Randy J. Chandler, Shawn M. Burgess, Mark E. Haskins, Margherita Dell’Anno, Charles H. Vite, Patricia O'Donnell, Marialuisa Alliegro, Edoardo Nusco
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 247-257 (2021)
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://explore.openaire.eu/search/publication?articleId=doi_dedup___::35a41d06530d415300146cd164955c67
http://europepmc.org/articles/PMC7803627
http://europepmc.org/articles/PMC7803627
Autor:
Margherita Dell’Anno, Alberto Auricchio, Stefania Galimberti, Edoardo Nusco, Rita Ferla, Jean-Brice Marteau, Vincent Zuliani, Maria Grazia Valsecchi, Marialuisa Alliegro, Severine Pouillot
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 143-158 (2017)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
In vivo gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. We recently demonstrated that AAV8-mediated liver gene transfer is effective in animal models of mucopolysaccharidosis type VI (MPS VI), a rare lysosomal
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c45e3363c2f12f3ebbea17952ef8cc46
http://www.sciencedirect.com/science/article/pii/S2329050117300876
http://www.sciencedirect.com/science/article/pii/S2329050117300876
Publikováno v:
Human gene therapy. 30(10)
Inborn errors of metabolism (IEM) are disorders affecting human biochemical pathways and represent attractive targets for gene therapy because of their severity, high overall prevalence, lack of effective treatments, and possibility of early diagnosi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a0c32744e13353462f9455aeff7facd9
https://pubmed.ncbi.nlm.nih.gov/31517544
https://pubmed.ncbi.nlm.nih.gov/31517544
Autor:
Giancarlo Parenti, Karen Kozarsky, John J. Hopwood, Rita Ferla, Generoso Andria, Alberto Auricchio, Ans T. van der Ploeg, Rossella Parini, Marco Savarese, Maurizio Scarpa, Hatice Serap Sivri, Vincenzo Nigro, Pamela Claudiani, Nicola Brunetti-Pierri, Maria Alice Donati, Giovanni Sorge, Simona Fecarotta
Publikováno v:
Human Gene Therapy
Human Gene Therapy, 26(3), 145-152. Mary Ann Liebert Inc.
Human Gene Therapy, 26(3), 145-152. Mary Ann Liebert Inc.
Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::43f387a870106574d90a0027e2392059
http://europepmc.org/articles/PMC4367235
http://europepmc.org/articles/PMC4367235
Autor:
Marialuisa Alliegro, Carmine Settembre, Rita Ferla, Edoardo Nusco, Chiara De Leonibus, Alberto Auricchio
Publikováno v:
Molecular Therapy
Enzyme replacement therapy (ERT) is the standard of care for several lysosomal storage diseases (LSDs). ERT, however, requires multiple and costly administrations and has limited efficacy. We recently showed that a single high dose administration of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3978c39ae7f7e8107e70eb4d41350312
http://hdl.handle.net/11588/651507
http://hdl.handle.net/11588/651507
