HK1237261B - 用於治疗威尔森病的核酸构建体和基因治疗载体 - Google Patents

用於治疗威尔森病的核酸构建体和基因治疗载体

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Publication number
HK1237261B
HK1237261B HK17111164.8A HK17111164A HK1237261B HK 1237261 B HK1237261 B HK 1237261B HK 17111164 A HK17111164 A HK 17111164A HK 1237261 B HK1237261 B HK 1237261B
Authority
HK
Hong Kong
Prior art keywords
atp7b
nucleic acid
hma
aav
aat
Prior art date
Application number
HK17111164.8A
Other languages
German (de)
English (en)
French (fr)
Other versions
HK1237261A1 (zh
Inventor
Oihana MURILLO SAUCA
Gloria GONZÁLEZ ASEGUINOLAZA
Rubén HERNÁNDEZ ALCOCEBA
Original Assignee
Fundación Para La Investigación Mèdica Aplicada
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Fundación Para La Investigación Mèdica Aplicada filed Critical Fundación Para La Investigación Mèdica Aplicada
Publication of HK1237261A1 publication Critical patent/HK1237261A1/zh
Publication of HK1237261B publication Critical patent/HK1237261B/zh

Links

Abstract

本发明涉及核酸建构和基因治疗载体那个包括ATP7B变体用于治疗与缺陷或功能障碍的铜运输ATPase 2，尤其是Wilson氏病。根据本发明设计的一个AAV矢量显著降低的尿铜的排泄，且肝Cu含量在肝豆状核变性小鼠进行矢量时，血清铜蓝蛋白活性显著恢复。另一方面，管理向量结果的归一化'血清转氨酶水平以及肝组织学，以及显著降低炎性渗液。
HK17111164.8A 2014-12-17 2015-12-17 用於治疗威尔森病的核酸构建体和基因治疗载体 HK1237261B (zh)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP14382531.3 2014-12-17

Publications (2)

Publication Number Publication Date
HK1237261A1 HK1237261A1 (zh) 2018-04-13
HK1237261B true HK1237261B (zh) 2021-06-25

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