Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients-Reference-Cited by-同舟云学术

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

Published:2021-03 Issue: Volume:20 Page:287-297
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Shi Xiaoxia,Aronson Sem J.,ten Bloemendaal Lysbeth,Duijst Suzanne,Bakker Robert S.,de Waart Dirk R.,Bortolussi Giulia,Collaud Fanny,Oude Elferink Ronald P.,Muro Andrés F.,Mingozzi Federico,Ronzitti Giuseppe,Bosma Piter J.

Funder

ZonMw

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

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5. Current therapy for Crigler-Najjar syndrome type 1: report of a world registry;van der Veere;Hepatology,1996

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