Zobrazeno 1 - 10
of 14
pro vyhledávání: '"Alessia De Caneva"'
Autor:
Giulia Bortolussi, Alessandra Iaconcig, Giulia Canarutto, Fabiola Porro, Filippo Ferrucci, Claudia Galletta, Cristian Díaz-Muñoz, Vipin Rawat, Alessia De Caneva, Olayemi Joseph Olajide, Lorena Zentilin, Silvano Piazza, Luka Bočkor, Andrés Fernando Muro
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101161- (2023)
(AAV)-mediated episomal gene replacement therapy for monogenic liver disorders is currently limited in pediatric settings due to the loss of vector DNA, associated with hepatocyte duplication during liver growth. Genome editing is a promising strateg
Externí odkaz:
https://doaj.org/article/c2f7c2a2a8134efd9f4b7723afb269fe
Autor:
Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon, Maria Grazia Biferi, Fabiola Porro, Adi Barzel, Lorena Zentilin, Mark A. Kay, Federico Mingozzi, Andrés F. Muro
Publikováno v:
Frontiers in Genome Editing, Vol 4 (2022)
Many inborn errors of metabolism require life-long treatments and, in severe conditions involving the liver, organ transplantation remains the only curative treatment. Non-integrative AAV-mediated gene therapy has shown efficacy in adult patients. Ho
Externí odkaz:
https://doaj.org/article/8b673b0e33fa4dbfbfb0304e87ef4951
Autor:
Fabiola Porro, Giulia Bortolussi, Adi Barzel, Alessia De Caneva, Alessandra Iaconcig, Simone Vodret, Lorena Zentilin, Mark A Kay, Andrés F Muro
Publikováno v:
EMBO Molecular Medicine, Vol 9, Iss 10, Pp 1346-1355 (2017)
Abstract Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and death. Liver transplantation is the only curative option, wh
Externí odkaz:
https://doaj.org/article/b8d3c49fae594bd4a42a4c908eba7487
Publikováno v:
PLoS ONE, Vol 9, Iss 8, p e104816 (2014)
The Crigler-Najjar Syndrome Type I (CNSI) is a rare genetic disorder caused by mutations in the Ugt1a1 gene. It is characterized by unconjugated hyperbilirubinemia that may result in severe neurologic damage and death if untreated. To date, liver tra
Externí odkaz:
https://doaj.org/article/84d3aa8c9673442395aeb91b77fb38c5
Autor:
Alessandra Iaconcig, Fabiola Porro, Mark A. Kay, Andrés F. Muro, Alessia De Caneva, Lorena Zentilin, Simone Vodret, Adi Barzel, Giulia Bortolussi
Publikováno v:
EMBO Molecular Medicine
Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and death. Liver transplantation is the only curative option, which has s
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b21899e0d5748b51923a31ee23c97976
https://pubmed.ncbi.nlm.nih.gov/28751579
https://pubmed.ncbi.nlm.nih.gov/28751579
Publikováno v:
PLoS ONE
PLoS ONE, Vol 9, Iss 8, p e104816 (2014)
PLoS ONE, Vol 9, Iss 8, p e104816 (2014)
The Crigler-Najjar Syndrome Type I (CNSI) is a rare genetic disorder caused by mutations in the Ugt1a1 gene. It is characterized by unconjugated hyperbilirubinemia that may result in severe neurologic damage and death if untreated. To date, liver tra
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::660c46b11b96c078db4de9de30c9dcd6
http://europepmc.org/articles/PMC4132024
http://europepmc.org/articles/PMC4132024
Autor:
Alessia De Caneva, Adi Barzel, Riccardo Sola, Giulia Bortolussi, Andrés F. Muro, Lorena Zentilin, Michela Lisjak, Mauro Giacca, Fabiola Porro, Mark A. Kay, Kristian Vlahoviček
Publikováno v:
Web of Science
Nonintegrative AAV-mediated gene therapy in the liver is effective in adult patients but faces limitations in pediatric settings because of episomal DNA loss during hepatocyte proliferation. Gene targeting is a promising approach as it results in the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d4931aa56e16e5acd188e912f5ecadba
https://publons.com/publon/53035870/
https://publons.com/publon/53035870/
Autor:
Infantino, Maria1 (AUTHOR), Tsalouchos, Aris2 (AUTHOR), Russo, Edda3 (AUTHOR), Laudicina, Selene2 (AUTHOR), Grossi, Valentina1 (AUTHOR), Lari, Barbara1 (AUTHOR), Benucci, Maurizio4 (AUTHOR), Stacchini, Lorenzo5 (AUTHOR), Amedei, Amedeo3 (AUTHOR) amedeo.amedei@unifi.it, Casprini, Patrizia6 (AUTHOR), Villalta, Danilo7 (AUTHOR), Dattolo, Pietro Claudio2 (AUTHOR), Manfredi, Mariangela1 (AUTHOR)
Publikováno v:
International Journal of Molecular Sciences. Oct2022, Vol. 23 Issue 20, p12333-N.PAG. 12p.
Autor:
Porro, Fabiola1, Bockor, Luka1, De Caneva, Alessia1, Bortolussi, Giulia1, Muro, Andrés F.1 muro@icgeb.org
Publikováno v:
PLoS ONE. Aug2014, Vol. 9 Issue 8, p1-10. 10p.
Publikováno v:
Gene Therapy Weekly; 12/18/2023, p2434-2434, 1p