Zobrazeno 1 - 10
of 120
pro vyhledávání: '"Piter J, Bosma"'
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:
Xiaoxia Shi, Giulia Bortolussi, Lysbeth Ten Bloemendaal, Suzanne Duijst, Andrés F Muro, Piter J Bosma
Publikováno v:
PLoS ONE, Vol 16, Iss 4, p e0250605 (2021)
In contrast to AAV, Simian Virus 40 (rSV40) not inducing neutralizing antibodies (NAbs) allowing re-treatment seems a promising vector for neonatal treatment of inherited liver disorders. Several studies have reported efficacy of rSV40 in animal mode
Externí odkaz:
https://doaj.org/article/a08eb17d84844a7a8088f691c132c137
Autor:
Xiaoxia Shi, Matthew Ryan Ykema, Jaco Hazenoot, Lysbeth ten Bloemendaal, Irene Mancini, Machteld Odijk, Peter de Haan, Piter J. Bosma
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 225-233 (2018)
Gene therapy has been shown to be a feasible approach to treat inherited disorders in vivo. Among the currently used viral vector systems, adeno-associated virus (AAV) vectors are the most advanced and have been applied in patients successfully. An i
Externí odkaz:
https://doaj.org/article/808dde88a3224fa88352b04ce933873e
Publikováno v:
BMC Pediatrics, Vol 19, Iss 1, Pp 1-3 (2019)
Abstract 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 pheno
Externí odkaz:
https://doaj.org/article/fd813391702a4386b7aa4d0dcf26a0f7
Autor:
Giulia Bortolussi, Xiaoxia Shi, Lysbeth ten Bloemendaal, Bhaswati Banerjee, Dirk R. De Waart, Gabriele Baj, Weiyu Chen, Ronald P. Oude Elferink, Ulrich Beuers, Coen C. Paulusma, Roland Stocker, Andrés F. Muro, Piter J. Bosma
Publikováno v:
Antioxidants, Vol 10, Iss 12, p 2029 (2021)
Accumulation of neurotoxic bilirubin due to a transient neonatal or persistent inherited deficiency of bilirubin glucuronidation activity can cause irreversible brain damage and death. Strategies to inhibit bilirubin production and prevent neurotoxic
Externí odkaz:
https://doaj.org/article/9e009047432a43d0942b9885748f455b
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
Autor:
Jyoti Naik, Chi M Hau, Lysbeth Ten Bloemendaal, Kam S Mok, Najat Hajji, Ann M Wehman, Sander Meisner, Vanesa Muncan, Nanne J Paauw, H E de Vries, Rienk Nieuwland, Coen C Paulusma, Piter J Bosma
Publikováno v:
PLoS ONE, Vol 14, Iss 4, p e0213069 (2019)
Extracellular vesicles (EVs) released by cells have a role in intercellular communication to regulate a wide range of biological processes. Two types of EVs can be recognized. Exosomes, which are released from multi-vesicular bodies upon fusion with
Externí odkaz:
https://doaj.org/article/f39e50ad0eb44039b02876af2f96ad43
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