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For treating the nucleic acid construct and gene therapy vector of Wilson disease
专利权人:
Fundacion para la Investigacion Medica Aplicada
发明人:
Oihana MURILLO SAUCA,Gloria GONZALEZ ASEGUINOLAZA,Ruben HERNANDEZ ALCOCEBA
申请号:
IL25291717
公开号:
IL252917D0
申请日:
2017.06.14
申请国别(地区):
IL
年份:
2017
代理人:
摘要:
The present invention relates to for treating illness, the especially nucleic acid construct and gene therapy vector comprising ATP7B variants of Wilson disease as caused by copper transhipment ATPase2 shortages or dysfunction. The AAV carriers designed according to the present invention significantly reduce urine Cu discharges and liver Cu contents in the Wilson disease mouse of the vehicle treated, while significant recovery ceruloplasmin activity. On the other hand, applying the carrier leads to serum aminotransferase levels at commencement and liver histological normalization, while being substantially reduced inflammatory infiltration.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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