Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Robert S, Bakker"'
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:
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
Autor:
Sem J. Aronson, Ulrich Beuers, Piter J. Bosma, Coen C. Paulusma, Xiaoxia Shi, Lysbeth ten Bloemendaal, Joanne Verheij, Suzanne Duijst, Giuseppe Ronzitti, Ronald P.J. Oude Elferink, Robert S. Bakker, Dirk R. de Waart
Publikováno v:
J Hepatol
J Hepatol, 2019, 71, pp.153-162. ⟨10.1016/j.jhep.2019.03.021⟩
Journal of hepatology, 71(1), 153-162. Elsevier
J Hepatol, 2019, 71, pp.153-162. ⟨10.1016/j.jhep.2019.03.021⟩
Journal of hepatology, 71(1), 153-162. Elsevier
International audience; BACKGROUND & AIMS: Progressive familial intrahepatic cholestasis type 3 (PFIC3), for which there are limited therapeutic options, often leads to end-stage liver disease before adulthood due to impaired ABCB4-dependent phosphol
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ac61c4989e9e887dc1d1403447b87ac5
https://pubmed.ncbi.nlm.nih.gov/30935993
https://pubmed.ncbi.nlm.nih.gov/30935993