[Nature] The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial

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connie0822 发表于 2026-7-28 15:59:59 | 显示全部楼层 |阅读模式
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期刊:Nature Medicine

文献作者:Jorg F. Hipp; Carlos A. Bacino; Lynne M. Bird; Ina Bruenig-Traebert; Daniel Chan; Marie Claire de Wit; Paulo Fontoura; Gregory Hooper; Ravi Jagasia; Michelle L. Krishnan; Lorraine Murtagh; Alessandro Noci; Ana Roche Martínez; Dietmar Schwab; Mercedes Serrano; Mark D. Shen; Julian Tillmann; Jorrit Tjeertes; Brenda Vincenzi; Elizabeth Berry-Kravis; Azad Bonni;  ; Muhamed Barakovic; Charlotte Bon; Ponghatai Boonsimma; Mariana Bustamante; Katalin Buzasi; Stormy Chamberlain; Susanne Clinch; Siobhan Connor-Ahmad; Philipp Daetwyler; Oana Danila; Sebastian Holst; Thomas Kremer; Florian Lipsmeier; Stefano Magon; Amanda Messer; David Nobbs; Giuseppe Palermo; Lisa Squassante; Thomas Wiese;  ; Jennifer M. Bain; Jamie Capal; Ralitza Gavrilova; María Munoz Cabeza; Nicola Pietrafusa; Rajsekar Rajamaran; Shafali Jeste; Federico Vigevano

出版日期:2025-9-

DOI号:10.1038/s41591-025-03784-7

下载链接:https://www.nature.com/articles/s41591-025-03784-7.pdf

文献链接:https://doi.org/10.1038/s41591-025-03784-7

当前文献来源于 Springer Science and Business Media LLC


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