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SAFE DELIVERY OF CRISPR AND OTHER GENE THERAPIES TO LARGE FRACTIONS OF SOMATIC CELLS IN HUMANS AND ANIMALS
专利权人:
Brian P. Hanley
发明人:
Brian P. Hanley
申请号:
US16097886
公开号:
US20200325483A1
申请日:
2017.05.12
申请国别(地区):
US
年份:
2020
代理人:
摘要:
A method for making it possible to deliver nucleic acid sequences to a large fraction of, or even 99.9% and more of the cells in a human body without near certainty of killing the recipient. It can be applied to safely deliver any gene therapy. This invention comprises a set of known compounds, many of them already approved, combined in novel ways to prevent immune system reaction to levels of delivery vehicle (capsid or synthetic carrier) introduced into the body that can be 5 or more orders of magnitude higher than has been demonstrated to cause human death. When used in concert with the disclosed CRISPR expression control method, this method can improve expression and allow better control over the gene therapy's target activity.
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中国工程科技知识中心
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http://www.ckcest.cn/home/

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