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VECTORS AND METHODS OF USE
专利权人:
Children's Hospitals and Clinics of Minnesota
发明人:
Joseph LILLEGARD
申请号:
US16775918
公开号:
US20200216823A1
申请日:
2020.01.29
申请国别(地区):
US
年份:
2020
代理人:
摘要:
This disclosure provides vectors and strategies for increasing the efficiency of gene therapy in hepatocytes. The efficiency of the delivery of a corrected gene or wild type gene is improved through the use of delivery to hepatocytes via intrahepatic (parenchyma) administration or administration via the portal vein. In addition, the corrected gene or wild type gene is delivered using an isolated exogenous nucleic acid comprising a promoter that is specifically expressed in hepatocytes. These methods and isolated exogenous nucleic acids are useful to correct gene defects in the liver such as inherited diseases of the liver.
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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