[EN] COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON DISEASE BY THE SELECTIVE DELIVERY OF OLIGONUCLEOTIDE MOLECULES TO SPECIFIC NEURON TYPES<br/>[FR] COMPOSITIONS ET MÉTHODES POUR LE TRAITEMENT DE LA MALADIE DE PARKINSON PAR ADMINISTRATION SÉLECTIVE DE MOLÉCULES D'OLIGONUCLÉOTIDES À DES TYPES SPÉCIFIQUES DE NEURONES
申请人:NLIFE THERAPEUTICS S L
公开号:WO2014064257A1
公开(公告)日:2014-05-01
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 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 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.