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Gene Therapy in Patients with the Crigler-Najjar Syndrome.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. D'Antiga L, et al. Among authors: bosma p. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. N Engl J Med. 2023. PMID: 37585628 Clinical Trial.
The phospholipid flippase ATP8B1 is involved in the pathogenesis of Ulcerative Colitis via establishment of intestinal barrier function.
Koelink PJ, Gómez-Mellado VE, Duijst S, van Roest M, Meisner S, Ho-Mok KS, Frank S, Appelman BS, Ten Bloemendaal L, Vogel GF, van de Graaf SFJ, Bosma PJ, Oude Elferink RPJ, Wildenberg ME, Paulusma CC. Koelink PJ, et al. Among authors: bosma pj. J Crohns Colitis. 2024 Feb 16:jjae024. doi: 10.1093/ecco-jcc/jjae024. Online ahead of print. J Crohns Colitis. 2024. PMID: 38366839
What's next in gene therapy for Crigler-Najjar syndrome?
Aronson SJ, Ronzitti G, Bosma PJ. Aronson SJ, et al. Among authors: bosma pj. Expert Opin Biol Ther. 2023 Feb;23(2):119-121. doi: 10.1080/14712598.2022.2160237. Epub 2022 Dec 29. Expert Opin Biol Ther. 2023. PMID: 36579791 No abstract available.
Disease burden and management of Crigler-Najjar syndrome: Report of a world registry.
Aronson SJ, Junge N, Trabelsi M, Kelmemi W, Hubert A, Brigatti KW, Fox MD, de Knegt RJ, Escher JC, Ginocchio VM, Iorio R, Zhu Y, Özçay F, Rahim F, El-Shabrawi MHF, Shteyer E, Di Giorgio A, D'Antiga L, Mingozzi F, Brunetti-Pierri N, Strauss KA, Labrune P, Mrad R, Baumann U, Beuers U, Bosma PJ; CureCN Consortium. Aronson SJ, et al. Among authors: bosma pj. Liver Int. 2022 Jul;42(7):1593-1604. doi: 10.1111/liv.15239. Epub 2022 Mar 29. Liver Int. 2022. PMID: 35274801 No abstract available.
Therapeutic base editing in the adult liver.
Paulusma CC, Bosma PJ. Paulusma CC, et al. Among authors: bosma pj. Nat Rev Gastroenterol Hepatol. 2021 Sep;18(9):597-598. doi: 10.1038/s41575-021-00491-9. Nat Rev Gastroenterol Hepatol. 2021. PMID: 34285418 No abstract available.
Reduced spontaneous itch in mouse models of cholestasis.
Langedijk J, Bolier R, Tolenaars D, Ten Bloemendaal L, Duijst S, de Waart D, Beuers U, Bosma P, Elferink RO. Langedijk J, et al. Among authors: bosma p. Sci Rep. 2021 Mar 17;11(1):6127. doi: 10.1038/s41598-021-85660-1. Sci Rep. 2021. PMID: 33731871 Free PMC article.
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Shi X, Aronson SJ, Ten Bloemendaal L, Duijst S, Bakker RS, de Waart DR, Bortolussi G, Collaud F, Oude Elferink RP, Muro AF, Mingozzi F, Ronzitti G, Bosma PJ. Shi X, et al. Among authors: bosma pj. Mol Ther Methods Clin Dev. 2020 Dec 3;20:287-297. doi: 10.1016/j.omtm.2020.11.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511243 Free PMC article.
115 results