Search Results - "De Caneva, Alessia"

Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model by Porro, Fabiola, Bortolussi, Giulia, Barzel, Adi, De Caneva, Alessia, Iaconcig, Alessandra, Vodret, Simone, Zentilin, Lorena, Kay, Mark A, Muro, Andrés F

“…Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of…”
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CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice by Bortolussi, Giulia, Iaconcig, Alessandra, Canarutto, Giulia, Porro, Fabiola, Ferrucci, Filippo, Galletta, Claudia, Díaz-Muñoz, Cristian, Rawat, Vipin, De Caneva, Alessia, Olajide, Olayemi Joseph, Zentilin, Lorena, Piazza, Silvano, Bočkor, Luka, Muro, Andrés Fernando

“…(AAV)-mediated episomal gene replacement therapy for monogenic liver disorders is currently limited in pediatric settings due to the loss of vector DNA,…”
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Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice by Lisjak, Michela, De Caneva, Alessia, Marais, Thibaut, Barbon, Elena, Biferi, Maria Grazia, Porro, Fabiola, Barzel, Adi, Zentilin, Lorena, Kay, Mark A, Mingozzi, Federico, Muro, Andrés F

“…Many inborn errors of metabolism require life-long treatments and, in severe conditions involving the liver, organ transplantation remains the only curative…”
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Generation of Ugt1-deficient murine liver cell lines using TALEN technology by Porro, Fabiola, Bockor, Luka, De Caneva, Alessia, Bortolussi, Giulia, Muro, Andrés F

“…The Crigler-Najjar Syndrome Type I (CNSI) is a rare genetic disorder caused by mutations in the Ugt1a1 gene. It is characterized by unconjugated…”
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Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases by De Caneva, Alessia, Porro, Fabiola, Bortolussi, Giulia, Sola, Riccardo, Lisjak, Michela, Barzel, Adi, Giacca, Mauro, Kay, Mark A, Vlahoviček, Kristian, Zentilin, Lorena, Muro, Andrés F

“…Non-integrative AAV-mediated gene therapy in the liver is effective in adult patients, but faces limitations in pediatric settings due to episomal DNA loss…”
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