Oligonucleotides for tissue specific gene expression modulation
AssigneeUniversity of Massachusetts
InventorsAnastasia Khvorova, Chantal Ferguson
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Abstract
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.
Claims47
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 an off-target tissue.
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A method of treating, suppressing, or reducing the severity of a neurodegenerative disease in a subject, the method comprising administering to the subject a therapeutic combination of drugs, the combination comprising:
- a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to a 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 tissue outside the central nervous system, to block the action of the first conjugate outside the central nervous system.
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A kit for the treatment or management of a neurodegenerative disease, the kit comprising:
- a first pharmaceutically acceptable composition of a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to a central nervous system,
- a second pharmaceutically acceptable composition of a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a tissue outside the central nervous system, and
- instructions for the administration of the first composition and second composition for treatment of a neurodegenerative disease.
A method of treating, suppressing, or reducing the severity of a neurodegenerative disease in a subject, the method comprising administering to the subject a therapeutically effective amount of an RNA silencing agent inhibiting the expression of a gene in a central nervous system, the improvement comprising administering to the subject a conjugate comprising an antagonist of the RNA silencing agent and a targeting agent that targets the conjugate to a liver, to selectively inhibit the RNA silencing agent in the liver.
A combination comprising:
- a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to a target tissue, and
- a second conjugate comprising an anti-RNA silencing agent and a second targeting agent that targets the second conjugate to an off-target tissue.
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Dependencies are inferred from the claim text.