Zobrazeno 1 - 10
of 23
pro vyhledávání: '"Sem J Aronson"'
Autor:
Xiaoxia Shi, Sem J. Aronson, Lysbeth ten Bloemendaal, Suzanne Duijst, Robert S. Bakker, Dirk R. de Waart, Giulia Bortolussi, Fanny Collaud, Ronald P. Oude Elferink, Andrés F. Muro, Federico Mingozzi, Giuseppe Ronzitti, Piter J. Bosma
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 287-297 (2021)
A clinical trial using adeno-associated virus serotype 8 (AAV8)-human uridine diphosphate glucuronosyltransferase 1A1 (hUGT1A1) to treat inherited severe unconjugated hyperbilirubinemia (Crigler-Najjar syndrome) is ongoing, but preclinical data sugge
Externí odkaz:
https://doaj.org/article/5f312cc89674405188672f6d384e4ef1
Autor:
Sem J. Aronson, Robert S. Bakker, Sascha Moenis, Remco van Dijk, Giulia Bortolussi, Fanny Collaud, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal, Giuseppe Ronzitti, Andrés F. Muro, Federico Mingozzi, Ulrich Beuers, Piter J. Bosma
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 250-258 (2020)
Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing authorization. We have developed and validated a cell-based, quantitative potency assay that detec
Externí odkaz:
https://doaj.org/article/edb7a29586e044c8b082be4c5aa3a2dd
Autor:
Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 157-174 (2019)
Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term efficacy of this approach in humans, showing efficient targeting of hepat
Externí odkaz:
https://doaj.org/article/721441a735dc4663ac629a4a43fbb03b
Autor:
Remco van Dijk, Sem J. Aronson, Dirk R. de Waart, Stan F. van de Graaf, Suzanne Duijst, Jurgen Seppen, Ronald Oude Elferink, Ulrich Beuers, Piter J. Bosma
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-9 (2017)
Abstract We aimed to identify potent biliverdin reductase (BVRA) inhibitors as a novel concept for the treatment of severe unconjugated hyperbilirubinemia. 1280 FDA-approved compounds were screened in vitro for their ability to inhibit human and rat
Externí odkaz:
https://doaj.org/article/d12f4548a8564f6a914ed21b89937903
Publikováno v:
Expert opinion on biological therapy. Taylor and Francis Ltd.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2ca245dd8779a26ad8775659940c679
https://pubmed.ncbi.nlm.nih.gov/36579791
https://pubmed.ncbi.nlm.nih.gov/36579791
Autor:
Piter J. Bosma, Andrés F. Muro, Giulia Bortolussi, Xiaoxia Shi, Sem J. Aronson, Dirk R. de Waart, Ronald P.J. Oude Elferink, Suzanne Duijst, Fanny Collaud, Robert S. Bakker, Lysbeth ten Bloemendaal, Giuseppe Ronzitti, Federico Mingozzi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 287-297 (2021)
Molecular therapy. Methods & clinical development, 20, 287-297. Nature Publishing Group
Molecular Therapy. Methods & Clinical Development
Molecular therapy. Methods & clinical development, 20, 287-297. Nature Publishing Group
Molecular Therapy. Methods & Clinical Development
A clinical trial using adeno-associated virus serotype 8 (AAV8)-human uridine diphosphate glucuronosyltransferase 1A1 (hUGT1A1) to treat inherited severe unconjugated hyperbilirubinemia (Crigler-Najjar syndrome) is ongoing, but preclinical data sugge
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::aee63f1483121a3b8be1553a09080dfd
http://www.scopus.com/inward/record.url?scp=85099261102&partnerID=8YFLogxK
http://www.scopus.com/inward/record.url?scp=85099261102&partnerID=8YFLogxK
Autor:
Giulia Bortolussi, Giuseppe Ronzitti, Lysbeth ten Bloemendaal, Piter J. Bosma, Andrés F. Muro, Robert S. Bakker, Xiaoxia Shi, Remco van Dijk, Federico Mingozzi, Fanny Collaud, Suzanne Duijst, Sem J. Aronson, Ulrich Beuers, Sascha Moenis
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 250-258 (2020)
Molecular Therapy-Methods & Clinical Development
Molecular therapy. Methods & clinical development, 18, 250-258. Nature Publishing Group
Molecular Therapy-Methods & Clinical Development
Molecular therapy. Methods & clinical development, 18, 250-258. Nature Publishing Group
Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing authorization. We have developed and validated a cell-based, quantitative potency assay that detec
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7ffb2207bb4b88df05c15b06a9c6750b
http://www.scopus.com/inward/record.url?scp=85086934701&partnerID=8YFLogxK
http://www.scopus.com/inward/record.url?scp=85086934701&partnerID=8YFLogxK
Autor:
Sem J, Aronson, Robert S, Bakker, Sascha, Moenis, Remco, van Dijk, Giulia, Bortolussi, Fanny, Collaud, Xiaoxia, Shi, Suzanne, Duijst, Lysbeth, Ten Bloemendaal, Giuseppe, Ronzitti, Andrés F, Muro, Federico, Mingozzi, Ulrich, Beuers, Piter J, Bosma
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing authorization. We have developed and validated a cell-based, quantitative potency assay that detec
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::fa333c5ac4f427366af7c8bb85ad0644
https://pubmed.ncbi.nlm.nih.gov/32637454
https://pubmed.ncbi.nlm.nih.gov/32637454
Publikováno v:
BMC Pediatrics, Vol 19, Iss 1, Pp 1-3 (2019)
BMC Pediatrics
BMC pediatrics, 19(1):173. BioMed Central
BMC Pediatrics
BMC pediatrics, 19(1):173. BioMed Central
Background Crigler-Najjar syndrome (CNs) presents as unconjugated hyperbilirubinemia, as a result of UGT1A1 deficiency, and can be categorized in a severe (type I) and mild (type II) phenotype. CNs type II patients usually benefit from phenobarbital
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e657eab0c70032101b33a98496c2a17e
http://link.springer.com/article/10.1186/s12887-019-1555-y
http://link.springer.com/article/10.1186/s12887-019-1555-y
Autor:
Sem J. Aronson, Angelo Di Giorgio, Ulrich Baumann, Virginie Delahais, Lorenzo D'Antiga, Fanny Collaud, Aurélie Hubert, Géraldine Honnet, Federico Mingozzi, Nicola Brunetti-Pierri, Philippe Labrune, Norman Junge, Robert J. de Knegt, Ulrich Beuers, Virginia Maria Ginocchio, Piter J. Bosma, Philippe Veron
Publikováno v:
Human Gene Therapy
Human Gene Therapy, Mary Ann Liebert, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human gene therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Human Gene Therapy, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human Gene Therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Hum Gene Ther
Hum Gene Ther, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human Gene Therapy, Mary Ann Liebert, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human gene therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Human Gene Therapy, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human Gene Therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Hum Gene Ther
Hum Gene Ther, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
International audience; Adeno-associated virus (AAV) vector-mediated gene therapy is currently evaluated as a potential treatment for Crigler-Najjar syndrome (CN) (NCT03466463). Pre-existing immunity to AAV is known to hinder gene transfer efficacy,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c1a2d9c9f9b11cded8a89710f8f8580
https://pubmed.ncbi.nlm.nih.gov/31502485
https://pubmed.ncbi.nlm.nih.gov/31502485