18675023. OLIGONUCLEOTIDES FOR TISSUE SPECIFIC GENE EXPRESSION MODULATION (University of Massachusetts)

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OLIGONUCLEOTIDES FOR TISSUE SPECIFIC GENE EXPRESSION MODULATION

Organization Name

University of Massachusetts

Inventor(s)

Anastasia Khvorova of Westborough MA (US)

Chantal Ferguson of Worcester MA (US)

OLIGONUCLEOTIDES FOR TISSUE SPECIFIC GENE EXPRESSION MODULATION

This abstract first appeared for US patent application 18675023 titled 'OLIGONUCLEOTIDES FOR TISSUE SPECIFIC GENE EXPRESSION MODULATION



Original Abstract Submitted

This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.