Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

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 c...

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Hauptverfasser: MONTEFELTRO, ANDRES PABLO, ALVARADO, GABRIEL G, ARTIGAS PEREZ, FRANCESC, CARMONA OROZCO, MARIA DEL CARMEN, VILA BOVER, MIQUEL, BORTOLOZZI, ANALIA
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HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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creator MONTEFELTRO, ANDRES PABLO
ALVARADO, GABRIEL G
ARTIGAS PEREZ, FRANCESC
CARMONA OROZCO, MARIA DEL CARMEN
VILA BOVER, MIQUEL
BORTOLOZZI, ANALIA
description 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.
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subjects HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
title Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
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