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NUCLEIC ACID MOLECULES AND THEIR METHODS OF USE
专利权人:
CHILDREN'S HOSPITAL MEDICAL CENTER
发明人:
Dao PAN
申请号:
US16498408
公开号:
US20200063132A1
申请日:
2018.03.29
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Some embodiments of the invention include a nucleic acid molecule comprising natural nucleotides, non-natural nucleotides, an LNA which comprises one or more RNA core molecules, or an RNA molecule which comprises more than one RNA core molecule. Some embodiments of the invention include a nucleic acid molecule comprising an RNA molecule which comprises more than one RNA core molecule. Other embodiments of the invention include a nucleic acid molecule comprising a DNA molecule encoding the RNA molecule (e.g., vector or viral vector). Other embodiments include compositions or pharmaceutical compositions that comprise the nucleic acid molecule. Some embodiments of the invention comprise reducing miR-143 in a cell. Other embodiments of the invention include methods to deliver a protein across the BBB. Other embodiments include methods for treating disease (e.g., LSD, neuronopathic disease, neurodegenerative disease, Hurler syndrome, or MPS I). Additional embodiments of the invention are also discussed herein.
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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