What's next in gene therapy for Crigler-Najjar syndrome?
Aronson, Sem J, Ronzitti, Giuseppe, Bosma, Piter J
Published in Expert opinion on biological therapy (01.02.2023)
Published in Expert opinion on biological therapy (01.02.2023)
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Journal Article
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
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
Published in Human gene therapy (01.10.2019)
Published in Human gene therapy (01.10.2019)
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Journal Article
Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice
Aronson, Sem J., Bakker, Robert S., Shi, Xiaoxia, Duijst, Suzanne, ten Bloemendaal, Lysbeth, de Waart, Dirk R., Verheij, Joanne, Ronzitti, Giuseppe, Oude Elferink, Ronald P., Beuers, Ulrich, Paulusma, Coen C., Bosma, Piter J.
Published in Journal of hepatology (01.07.2019)
Published in Journal of hepatology (01.07.2019)
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Journal Article
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
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
Published in Molecular therapy. Methods & clinical development (15.03.2019)
Published in Molecular therapy. Methods & clinical development (15.03.2019)
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Journal Article
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
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.
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Journal Article
Biliverdin Reductase inhibitors did not improve severe unconjugated hyperbilirubinemia in vivo
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
Published in Scientific reports (10.05.2017)
Published in Scientific reports (10.05.2017)
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Journal Article
Disease burden and management of Crigler‐Najjar syndrome: Report of a world registry
Aronson, Sem J., Junge, Norman, Trabelsi, Mediha, Kelmemi, Wided, Hubert, Aurelie, Brigatti, Karlla W., Fox, Michael D., Knegt, Robert J., Escher, Johanna C., Ginocchio, Virginia M., Iorio, Raffaele, Zhu, Yan, Özçay, Figen, Rahim, Fakher, El‐Shabrawi, Mortada H.F., Shteyer, Eyal, Di Giorgio, Angelo, D'Antiga, Lorenzo, Mingozzi, Federico, Brunetti‐Pierri, Nicola, Strauss, Kevin A., Labrune, Philippe, Mrad, Ridha, Baumann, Ulrich, Beuers, Ulrich, Bosma, Piter J.
Published in Liver international (01.07.2022)
Published in Liver international (01.07.2022)
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Journal Article
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
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.
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Published in Molecular therapy. Methods & clinical development (11.09.2020)
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Journal Article
A novel UGT1A1 gene mutation causing severe unconjugated hyperbilirubinemia: a case report
Shi, Xiaoxia, Aronson, Sem, Khan, Ahmed Sharif, Bosma, Piter J
Published in BMC pediatrics (29.05.2019)
Published in BMC pediatrics (29.05.2019)
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Journal Article
Colonial History and Contemporary Transmission Shape the Genetic Diversity of Hepatitis C Virus Genotype 2 in Amsterdam
MARKOV, Peter V, DE LAAR, Thijs J. Van, THOMAS, Xiomara V, ARONSON, Sem J, WEEGINK, Christine J, DEN BERK, Guido E. Van, PRINS, Maria, PYBUS, Oliver G, SCHINKEL, Janke
Published in Journal of Virology (01.07.2012)
Published in Journal of Virology (01.07.2012)
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Journal Article
Emergence of Hepatitis C Virus Genotype 4: Phylogenetic Analysis Reveals Three Distinct Epidemiological Profiles
de Bruijne, Joep, Schinkel, Janke, Prins, Maria, Koekkoek, Sylvie M, Aronson, Sem J, van Ballegooijen, Marijn W, Reesink, Hendrik W, Molenkamp, Richard, van de Laar, Thijs J.W
Published in Journal of Clinical Microbiology (01.12.2009)
Published in Journal of Clinical Microbiology (01.12.2009)
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Journal Article
Disruption of HNF1α binding site causes inherited severe unconjugated hyperbilirubinemia
van Dijk, Remco, Mayayo-Peralta, Isabel, Aronson, Sem J, Kattentidt-Mouravieva, Anja A, van der Mark, Vincent A, de Knegt, Rob, Oruc, Nevin, Beuers, Ulrich, Bosma, Piter J
Published in Journal of hepatology (01.12.2015)
Published in Journal of hepatology (01.12.2015)
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Journal Article
Gene Therapy in Patients with the Crigler–Najjar Syndrome
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
Published in The New England journal of medicine (17.08.2023)
Published in The New England journal of medicine (17.08.2023)
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Journal Article
SARS-CoV-2 viral-load distribution reveals that viral loads increase with age: a retrospective cross-sectional cohort study
Euser, Sjoerd, Aronson, Sem, Manders, Irene, van Lelyveld, Steven, Herpers, Bjorn, Sinnige, Jan, Kalpoe, Jayant, van Gemeren, Claudia, Snijders, Dominic, Jansen, Ruud, Schuurmans Stekhoven, Sophie, van Houten, Marlies, Lede, Ivar, Cohen Stuart, James, Slijkerman Megelink, Fred, Kapteijns, Erik, den Boer, Jeroen, Sanders, Elisabeth, Wagemakers, Alex, Souverein, Dennis
Published in International journal of epidemiology (01.12.2021)
Published in International journal of epidemiology (01.12.2021)
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Journal Article
SARS-CoV-2 viral-load distribution reveals that viral loads increase with age: a retrospective cross-sectional cohort study
Euser, Sjoerd, Aronson, Sem, Manders, Irene, van Lelyveld, Steven, Herpers, Bjorn, Sinnige, Jan, Kalpoe, Jayant, van Gemeren, Claudia, Snijders, Dominic, Jansen, Ruud, Schuurmans Stekhoven, Sophie, van Houten, Marlies, Lede, Ivar, Cohen Stuart, James, Slijkerman Megelink, Fred, Kapteijns, Erik, den Boer, Jeroen, Sanders, Elisabeth, Wagemakers, Alex, Souverein, Dennis
Published in International Journal of Epidemiology (06.01.2022)
Published in International Journal of Epidemiology (06.01.2022)
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Journal Article
Web Resource
SARS-CoV-2 viral-load distribution reveals that viral loads increase with age: a retrospective cross-sectional cohort study
Euser, Sjoerd, Aronson, Sem, Manders, Irene, van Lelyveld, Steven, Herpers, Bjorn, Sinnige, Jan, Kalpoe, Jayant, van Gemeren, Claudia, Snijders, Dominic, Jansen, Ruud, Schuurmans Stekhoven, Sophie, van Houten, Marlies, Lede, Ivar, Cohen Stuart, James, Slijkerman Megelink, Fred, Kapteijns, Erik, den Boer, Jeroen, Sanders, Elisabeth, Wagemakers, Alex, Souverein, Dennis
Published in International journal of epidemiology (08.09.2021)
Published in International journal of epidemiology (08.09.2021)
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Journal Article
Initial Severe Acute Respiratory Syndrome Coronavirus 2 Viral Load Is Associated With Disease Severity: A Retrospective Cohort Study
Souverein, Dennis, van Stralen, Karlijn, van Lelyveld, Steven, van Gemeren, Claudia, Haverkort, Milly, Snijders, Dominic, Soetekouw, Robin, Kapteijns, Erik, de Jong, Evelien, Hermanides, Gonneke, Aronson, Sem, Herpers, Bjorn, den Boer, Jeroen, Wagemakers, Alex, Euser, Sjoerd
Published in Open forum infectious diseases (01.07.2022)
Published in Open forum infectious diseases (01.07.2022)
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Journal Article
Efficacy of AAV8-h UGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
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
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Published in Molecular therapy. Methods & clinical development (12.03.2021)
Journal Article
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem, Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine, Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia, Martin, Samia, Le Bec, Christine, Bosma, Piter, Muro, Andres, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Published in Molecular therapy. Methods & clinical development (01.03.2019)
Published in Molecular therapy. Methods & clinical development (01.03.2019)
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Journal Article
Thermolysis and Photolysis of C60 Diozonides
Heymann, Dieter, Bachilo, Sergei M., Aronson, Sem
Published in Fullerenes, nanotubes, and carbon nanostructures (01.01.2005)
Published in Fullerenes, nanotubes, and carbon nanostructures (01.01.2005)
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