Title: | A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome |
Journal : | Molecular therapy. Methods & clinical development, 3 |
Authors: | Ronzitti G, Author ; 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 |
Material Type: | Article |
Publication Date: | 2016 |
Languages: | English |
Keywords : | adenoassociated vectors ; Crigler-Najjar syndrome ; exon-intron structure ; gene expression ; gene therapy ; liver ; mouse ; preclinical trials ; rat ; UGT1A1 gene ; Western blot |
Pubmed / DOI : | Pubmed : 27722180 / DOI : 10.1038/mtm.2016.49 |
See also : |