发明名称 |
COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON DISEASE BY THE SELECTIVE DELIVERY OF OLIGONUCLEOTIDE MOLECULES TO SPECIFIC NEURON TYPES |
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), serotonin 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 interest 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. |
申请公布号 |
EP2911695(A1) |
申请公布日期 |
2015.09.02 |
申请号 |
EP20130783061 |
申请日期 |
2013.10.25 |
申请人 |
NLIFE THERAPEUTICS S.L. |
发明人 |
CARMONA OROZCO, MARIA DEL CARMEN;MONTEFELTRO, ANDRÉS PABLO;ALVARADO, GABRIEL G.;VILA BOVER, MIQUEL;BORTOLOZZI, ANALIA;ARTIGAS PÉREZ, FRANCESC |
分类号 |
A61K47/48;A61K31/7105;A61P25/16 |
主分类号 |
A61K47/48 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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