A novel UGT1A1 gene mutation causing severe unconjugated hyperbilirubinemia: a case report

por Shi, Xiaoxia, Aronson, Sem, Khan, Ahmed Sharif, Bosma, Piter J.
Publicado 2019

Gene Therapy for Progressive Familial Intrahepatic Cholestasis: Current Progress and Future Prospects

por Bosma, Piter J., Wits, Marius, Oude-Elferink, Ronald P. J.
Publicado 2020

Insulin-like growth factor binding protein 5 enhances survival of LX2 human hepatic stellate cells

por Sokolović, Aleksandar, Sokolović, Milka, Boers, Willem, Elferink, Ronald PJ Oude, Bosma, Piter J
Publicado 2010

Low efficacy of recombinant SV40 in Ugt1a1(-/-) mice with severe inherited hyperbilirubinemia

por Shi, Xiaoxia, Bortolussi, Giulia, Bloemendaal, Lysbeth ten, Duijst, Suzanne, Muro, Andrés F., Bosma, Piter J.
Publicado 2021

Adeno-Associated Viral Vector Serotype 5 Poorly Transduces Liver in Rat Models

por Montenegro-Miranda, Paula S., Pañeda, Astrid, ten Bloemendaal, Lysbeth, Duijst, Suzanne, de Waart, Dirk R., Aseguinolaza, Gloria Gonzalez, Bosma, Piter J.
Publicado 2013

Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40

por Shi, Xiaoxia, Ykema, Matthew Ryan, Hazenoot, Jaco, ten Bloemendaal, Lysbeth, Mancini, Irene, Odijk, Machteld, de Haan, Peter, Bosma, Piter J.
Publicado 2018

Biliverdin Reductase inhibitors did not improve severe unconjugated hyperbilirubinemia in vivo

por van Dijk, Remco, Aronson, Sem J., de Waart, Dirk R., van de Graaf, Stan F., Duijst, Suzanne, Seppen, Jurgen, Elferink, Ronald Oude, Beuers, Ulrich, Bosma, Piter J.
Publicado 2017

A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

por Aronson, Sem J., Bakker, Robert S., Moenis, Sascha, van Dijk, Remco, Bortolussi, Giulia, Collaud, Fanny, Shi, Xiaoxia, Duijst, Suzanne, ten Bloemendaal, Lysbeth, Ronzitti, Giuseppe, Muro, Andrés F., Mingozzi, Federico, Beuers, Ulrich, Bosma, Piter J.
Publicado 2020

The P4-ATPase ATP9A is a novel determinant of exosome release

por Naik, Jyoti, Hau, Chi M., ten Bloemendaal, Lysbeth, Mok, Kam S., Hajji, Najat, Wehman, Ann M., Meisner, Sander, Muncan, Vanesa, Paauw, Nanne J., de Vries, H. E., Nieuwland, Rienk, Paulusma, Coen C., Bosma, Piter J.
Publicado 2019

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

por Shi, Xiaoxia, Aronson, Sem J., ten Bloemendaal, Lysbeth, Duijst, Suzanne, Bakker, Robert S., de Waart, Dirk R., Bortolussi, Giulia, Collaud, Fanny, Oude Elferink, Ronald P., Muro, Andrés F., Mingozzi, Federico, Ronzitti, Giuseppe, Bosma, Piter J.
Publicado 2020

Long-Term Effects of Biliverdin Reductase a Deficiency in Ugt1(−/−) Mice: Impact on Redox Status and Metabolism

por Bortolussi, Giulia, Shi, Xiaoxia, ten Bloemendaal, Lysbeth, Banerjee, Bhaswati, De Waart, Dirk R., Baj, Gabriele, Chen, Weiyu, Oude Elferink, Ronald P., Beuers, Ulrich, Paulusma, Coen C., Stocker, Roland, Muro, Andrés F., Bosma, Piter J.
Publicado 2021

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

por Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Publicado 2016

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome

por Aronson, Sem J., Veron, Philippe, Collaud, Fanny, Hubert, Aurélie, Delahais, Virginie, Honnet, Géraldine, de Knegt, Robert J., Junge, Norman, Baumann, Ulrich, Di Giorgio, Angelo, D'Antiga, Lorenzo, Ginocchio, Virginia M., Brunetti-Pierri, Nicola, Labrune, Philippe, Beuers, Ulrich, Bosma, Piter J., Mingozzi, Federico
Publicado 2019

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

por Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem J., Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine G., Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia v., Martin, Samia, Le Bec, Christine, Bosma, Piter J., Muro, Andres F., Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Publicado 2018