Search Results - "Boffa, Iolanda"

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    Liver‐directed gene therapy for ornithine aminotransferase deficiency by Boffa, Iolanda, Polishchuk, Elena, De Stefano, Lucia, Dell'Aquila, Fabio, Nusco, Edoardo, Marrocco, Elena, Audano, Matteo, Pedretti, Silvia, Caterino, Marianna, Bellezza, Ilaria, Ruoppolo, Margherita, Mitro, Nico, Cellini, Barbara, Auricchio, Alberto, Brunetti‐Pierri, Nicola

    Published in EMBO molecular medicine (11-04-2023)
    “…Gyrate atrophy of choroid and retina (GACR) is a chorioretinal degeneration caused by pathogenic variants in the gene encoding ornithine aminotransferase…”
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    Journal Article
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    Vision on gyrate atrophy: why treat the liver? by Boffa, Iolanda, Brunetti-Pierri, Nicola

    Published in EMBO molecular medicine (16-01-2024)
    “…Gyrate Atrophy of the Choroid and Retina (GACR) currently lacks any effective treatment and a highly restricted, yet cumbersome, low-arginine diet is the only…”
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    Journal Article
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    Liver gene transfer for metabolite detoxification in inherited metabolic diseases by D'Alessio, Alfonso M., Boffa, Iolanda, De Stefano, Lucia, Soria, Leandro R., Brunetti‐Pierri, Nicola

    Published in FEBS letters (01-10-2024)
    “…Inherited metabolic disorders (IMDs) are a growing group of genetic diseases caused by defects in enzymes that mediate cellular metabolism, often resulting in…”
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    Journal Article
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    Natural compounds for pediatric cancer treatment by Ferrucci, Veronica, Boffa, Iolanda, De Masi, Gina, Zollo, Massimo

    “…There is a tremendous need in clinics to impair cancer progression through noninvasive therapeutic approaches. The use of natural compounds to achieve this is…”
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    Journal Article Book Review
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    CD19 Redirected CAR NK Cells Are Equally Effective but Less Toxic Than CAR T Cells by Quintarelli, Concetta, Sivori, Simona, Caruso, Simona, Carlomagno, Simona, Boffa, Iolanda, Orlando, Domenico, Guercio, Marika, Cembrola, Biancamaria, Pitisci, Angela, Di Cecca, Stefano, Li Pira, Giuseppina, Vinti, Luciana, De Angelis, Biagio, Moretta, Lorenzo, Locatelli, Franco

    Published in Blood (29-11-2018)
    “…Based on the clinical success observed in acute lymphoblastic leukemia (ALL) with chimeric antigen receptor engineered T (CAR T), we hypothesized that…”
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    Journal Article
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