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COMPOSITIONS AND METHODS FOR SELECTIVE DELIVERY OF OLIGONUCLEOTIDE MOLECULES TO SPECIFIC NEURON TYPES
专利权人:
MiCure Therapeutics Ltd
发明人:
MONTEFELTRO, Andrés Pablo,ALVARADO URBINA, Gabriel,BORTOLOZZI BIASSONI, Analia,ARTIGAS PÉREZ, Francesc,VILA BOVER, Miquel
申请号:
AU2020202533
公开号:
AU2020202533A1
申请日:
2020.04.15
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
#$%^&*AU2020202533A120200507.pdf#####ABSTRACT The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or 5 reduces expression of the target nucleic acid and (ii) a selectivity agent which is capable of binding with high affinity to a neurotransmitter transporter. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interests and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the 10 cells for diagnostic purposes.
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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