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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:
NLIFE THERAPEUTICS; S.L.
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:
CA2890112
公开号:
CA2890112A1
申请日:
2013.10.25
申请国别(地区):
CA
年份:
2014
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 a-synuclein or the mRNA encoding a-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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