Zobrazeno 1 - 10
of 33
pro vyhledávání: '"Lysbeth, Ten Bloemendaal"'
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:
Jacqueline Langedijk, Ruth Bolier, Dagmar Tolenaars, Lysbeth ten Bloemendaal, Suzanne Duijst, Dirk de Waart, Ulrich Beuers, Piter Bosma, Ronald Oude Elferink
Publikováno v:
Scientific Reports, Vol 11, Iss 1, Pp 1-13 (2021)
Abstract Pruritus is one of the most distressing symptoms in cholestatic patients. Plasma autotaxin (ATX) activity correlates with the severity of pruritus in cholestatic patients, but the pathophysiology is unclear. To study pruritus in mice, we mea
Externí odkaz:
https://doaj.org/article/8a91086451c548c488f3ed16188f6c17
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:
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
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:
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
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:
Piter J. Bosma, Giulia Bortolussi, Andrés F. Muro, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal
Publikováno v:
PLoS ONE, 16(4 April):e0250605. Public Library of Science
PLoS ONE
PLoS ONE, Vol 16, Iss 4, p e0250605 (2021)
PLoS ONE
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://explore.openaire.eu/search/publication?articleId=doi_dedup___::7cc82709d79be8fd923ebfb1c62e8ac2
https://pure.amc.nl/en/publications/low-efficacy-of-recombinant-sv40-in-ugt1a1-mice-with-severe-inherited-hyperbilirubinemia(73eff083-026d-4d0f-86ab-9924c9d3cf78).html
https://pure.amc.nl/en/publications/low-efficacy-of-recombinant-sv40-in-ugt1a1-mice-with-severe-inherited-hyperbilirubinemia(73eff083-026d-4d0f-86ab-9924c9d3cf78).html
