Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
申请人:Nlife Therapeutics S.L.
公开号:EP2380595A1
公开(公告)日:2011-10-26
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 nucleuc acid toi 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 contrast agents to the cells for diagnostic purposes.
本发明提供了一种共轭物,该共轭物包含(i)与靶核酸序列互补的核酸,该核酸的表达可防止或减少靶核酸的表达;(ii)能够与神经递质转运体高亲和力结合的选择剂。本发明的共轭物可用于将核酸递送至相关细胞,从而治疗需要下调靶核酸编码蛋白的疾病,也可用于将造影剂递送至细胞以进行诊断。