Gene Therapy in Patients with the Crigler–Najjar Syndrome von D’Antiga, Lorenzo, Beuers, Ulrich, Ronzitti, Giuseppe, Brunetti-Pierri, Nicola, Baumann, Ulrich, Di Giorgio, Angelo, Aronson, Sem, Hubert, Aurelie, Romano, Roberta, Junge, Norman, Bosma, Piter, Bortolussi, Giulia, Muro, Andrés F., Soumoudronga, Ravaka F., Veron, Philippe, Collaud, Fanny, Knuchel-Legendre, Nathalie, Labrune, Philippe, Mingozzi, Federico

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Bilirubin-Induced Oxidative Stress Leads to DNA Damage in the Cerebellum of Hyperbilirubinemic Neonatal Mice and Activates DNA Double-Strand Break Repair Pathways in Human Cells von Tiribelli, Claudio, Gazzin, Silvia, Bortolussi, Giulia, Rawat, Vipin, Muro, Andrés F.

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Experimental models assessing bilirubin neurotoxicity von Bortolussi, Giulia, Muro, Andrés F.

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Attenuation of neuro-inflammation improves survival and neurodegeneration in a mouse model of severe neonatal hyperbilirubinemia von Vodret, Simone, Bortolussi, Giulia, Iaconcig, Alessandra, Martinelli, Elena, Tiribelli, Claudio, Muro, Andrés F.

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Fibronectin splice variants: Understanding their multiple roles in health and disease using engineered mouse models von White, Eric S., Muro, Andrés F.

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Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia von Shi, Xiaoxia, Bortolussi, Giulia, Bloemendaal, Lysbeth Ten, Duijst, Suzanne, Muro, Andrés F, Bosma, Piter J

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An Essential Role for Fibronectin Extra Type III Domain A in Pulmonary Fibrosis von Muro, Andres F, Moretti, Federico A, Moore, Bethany B, Yan, Mei, Atrasz, Rachelle G, Wilke, Carol A, Flaherty, Kevin R, Martinez, Fernando J, Tsui, Jessica L, Sheppard, Dean, Baralle, Francisco E, Toews, Galen B, White, Eric S

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Inflammatory signature of cerebellar neurodegeneration during neonatal hyperbilirubinemia in Ugt1 -/- mouse model von Vodret, Simone, Bortolussi, Giulia, Jašprová, Jana, Vitek, Libor, Muro, Andrés F

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Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice von Tsuji, Shinnosuke, Stephens, Calvin J., Bortolussi, Giulia, Zhang, Feijie, Baj, Gabriele, Jang, Hagoon, de Alencastro, Gustavo, Muro, Andrés F., Pekrun, Katja, Kay, Mark A.

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

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Rescue of bilirubin‐induced neonatal lethality in a mouse model of Crigler‐Najjar syndrome type I by AAV9‐mediated gene transfer von Bortolussi, Giulia, Zentilin, Lorena, Baj, Gabriele, Giraudi, Pablo, Bellarosa, Cristina, Giacca, Mauro, Tiribelli, Claudio, Muro, Andrés F.

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Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9 von Lisjak, Michela, Iaconcig, Alessandra, Guarnaccia, Corrado, Vicidomini, Antonio, Moretti, Laura, Collaud, Fanny, Ronzitti, Giuseppe, Zentilin, Lorena, Muro, Andrés F.

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Formation of an invasion-permissive matrix requires TGFβ/SNAIL1-regulated alternative splicing of fibronectin von Franco-Valls, Héctor, Tusquets-Uxó, Elsa, Sala, Laura, Val, Maria, Peña, Raúl, Iaconcig, Alessandra, Villarino, Álvaro, Jiménez-Arriola, Martín, Massó, Pere, Trincado, Juan L, Eyras, Eduardo, Muro, Andrés F, Otero, Jorge, García de Herreros, Antonio, Baulida, Josep

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Integrin-α9 Is Required for Fibronectin Matrix Assembly during Lymphatic Valve Morphogenesis von Bazigou, Eleni, Xie, Sherry, Chen, Chun, Weston, Anne, Miura, Naoyuki, Sorokin, Lydia, Adams, Ralf, Muro, Andrés F., Sheppard, Dean, Makinen, Taija

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Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome von Bortolussi, Giulia, Zentillin, Lorena, Vaníkova, Jana, Bockor, Luka, Bellarosa, Cristina, Mancarella, Antonio, Vianello, Eleonora, Tiribelli, Claudio, Giacca, Mauro, Vitek, Libor, Muro, Andrés F

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Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice von 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

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TDP-43 regulates β-adducin (Add2) transcript stability von Costessi, Luisa, Porro, Fabiola, Iaconcig, Alessandra, Muro, Andrés F

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Generation of Ugt1-deficient murine liver cell lines using TALEN technology von Porro, Fabiola, Bockor, Luka, De Caneva, Alessia, Bortolussi, Giulia, Muro, Andrés F

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Recipient-derived EDA fibronectin promotes cardiac allograft fibrosis von Booth, Adam J, Wood, Sherri C, Cornett, Ashley M, Dreffs, Alyssa A, Lu, Guanyi, Muro, Andrés F, White, Eric S, Bishop, D Keith

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Brief Report: Alternative Splicing of Extra Domain A (EIIIA) of Fibronectin Plays a Tissue‐Specific Role in Hematopoietic Homeostasis von Malara, Alessandro, Gruppi, Cristian, Celesti, Giuseppe, Romano, Bina, Laghi, Luigi, Marco, Luigi, Muro, Andrés F., Balduini, Alessandra

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