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Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
专利权人:
S.L.;nLife Therapeutics
发明人:
Carmona Orozco, Maria del Carmen,Montefeltro, Andres Pablo,Alvarado, Gabriel G.,Vila Bover, Miquel,Bortolozzi, Analia,Artigas Perez, Francesc
申请号:
AU2013336581
公开号:
AU2013336581A1
申请日:
2013.10.25
申请国别(地区):
AU
年份:
2015
代理人:
摘要:
The invention provides a conjugate comprising(i) a selectivity agent which binds specifically to one or more neurotransmitter transporters selected from the group consisting of a dopamine transporter (DAT), serotonine transporter (SERT) or a norepinephrine transporter (NET) and (ii) a nucleic acid capable of specifically binding to a target molecule which is expressed in the same cell as the neurotransmitter transporter wherein said target molecule is α-synuclein or the mRNA encoding α-synuclein. 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 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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