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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. Ronzitti G, et al. Among authors: bosma pj. Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. doi: 10.1038/mtm.2016.49. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722180 Free PMC article.
In the rat liver, Adenoviral gene transfer efficiency is comparable to AAV.
Montenegro-Miranda PS, Pichard V, Aubert D, Ten Bloemendaal L, Duijst S, de Waart DR, Ferry N, Bosma PJ. Montenegro-Miranda PS, et al. Among authors: bosma pj. Gene Ther. 2014 Feb;21(2):168-74. doi: 10.1038/gt.2013.69. Epub 2013 Nov 28. Gene Ther. 2014. PMID: 24285217
Characterization and treatment of persistent hepatocellular secretory failure.
van Dijk R, Kremer AE, Smit W, van den Elzen B, van Gulik T, Gouma D, Lameris JS, Bikker H, Enemuo V, Stokkers PC, Feist M, Bosma P, Jansen PL, Beuers U. van Dijk R, et al. Liver Int. 2015 Apr;35(4):1478-88. doi: 10.1111/liv.12603. Epub 2014 Jul 4. Liver Int. 2015. PMID: 24905729
Gene replacement therapy for genetic hepatocellular jaundice.
van Dijk R, Beuers U, Bosma PJ. van Dijk R, et al. Among authors: bosma pj. Clin Rev Allergy Immunol. 2015 Jun;48(2-3):243-53. doi: 10.1007/s12016-014-8454-7. Clin Rev Allergy Immunol. 2015. PMID: 25315738 Review.
De Novo Donor-Specific HLA Antibody Formation in Two Patients With Crigler-Najjar Syndrome Type I Following Human Hepatocyte Transplantation With Partial Hepatectomy Preconditioning.
Jorns C, Nowak G, Nemeth A, Zemack H, Mörk LM, Johansson H, Gramignoli R, Watanabe M, Karadagi A, Alheim M, Hauzenberger D, van Dijk R, Bosma PJ, Ebbesen F, Szakos A, Fischler B, Strom S, Ellis E, Ericzon BG. Jorns C, et al. Among authors: bosma pj. Am J Transplant. 2016 Mar;16(3):1021-30. doi: 10.1111/ajt.13487. Epub 2015 Nov 2. Am J Transplant. 2016. PMID: 26523372 Free PMC article.
97 results