发明名称 |
COMPOSITIONS AND METHODS FOR SELECTIVE DELIVERY OF OLIGONUCLEOTIDE MOLECULES TO SPECIFIC NEURON TYPES |
摘要 |
The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or 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 cells for diagnostic purposes. |
申请公布号 |
KR20130103662(A) |
申请公布日期 |
2013.09.24 |
申请号 |
KR20127030202 |
申请日期 |
2011.04.19 |
申请人 |
NLIFE THERAPEUTICS S.L. |
发明人 |
MONTEFELTRO ANDRES PABLO;ALVARADO URBINA GABRIEL;BORTOLOZZI BIASSONI ANALIA;ARTIGAS PEREZ FRANCESC;VILA BOVER MIQUEL |
分类号 |
C12N15/11;A61K47/48;A61K48/00;A61K49/08 |
主分类号 |
C12N15/11 |
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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