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Atp8b1 deficiency in mice reduces resistance of the canalicular membrane to hydrophobic bile salts and impairs bile salt transport.
Paulusma CC, Groen A, Kunne C, Ho-Mok KS, Spijkerboer AL, Rudi de Waart D, Hoek FJ, Vreeling H, Hoeben KA, van Marle J, Pawlikowska L, Bull LN, Hofmann AF, Knisely AS, Oude Elferink RP. Paulusma CC, et al. Among authors: rudi de waart d. Hepatology. 2006 Jul;44(1):195-204. doi: 10.1002/hep.21212. Hepatology. 2006. PMID: 16799980
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
Shi X, Bortolussi G, Collaud F, Lebrun PR, Bloemendaal LT, Guerchet N, Rudi de Waart D, Sellier P, Duijst S, Veron P, Mingozzi F, Kishimoto TK, Ronzitti G, Bosma P, Muro AF. Shi X, et al. Among authors: rudi de waart d. Mol Ther Methods Clin Dev. 2024 Oct 28;32(4):101363. doi: 10.1016/j.omtm.2024.101363. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39618425 Free PMC article.