Patent ID: 9084825
Filing Date: 2015-07-21
Classification: A61K,A61P,C12N

Abstract:
1. A conjugate comprising (i) at least one selectivity agent which binds specifically to one or more neurotransmitter transporters selected from the group consisting of a dopamine transporter (DAT), a serotonin transporter (SERT), and a norepinephrine transporter (NET) and (ii) at least one nucleic acid which is 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; wherein the at least one selectivity agent and the at least one nucleic acid are covalently coupled, wherein the covalent coupling is either direct or via a linking group, wherein the at least one nucleic acid is a gapmer, wherein the gapmer comprises a central block of 10 deoxynucleotides flanked by blocks of 4 2′-O-methyl modified ribonucleotides, and wherein the gapmer consists of a sequence selected from the group consisting of SEQ ID NO:1, SEQ ID NO:2, and SEQ ID NO:3.