Compositions for inhibiting dipeptide repeat protein-ribosomal rna interaction and uses thereof
AssigneesNorthwestern University, The United States of America as represented by the Secretary, Department of Health and Human Service
InventorsSandra Wolin, Marco Boccitto, Juan Alberto Ortega Cano, Evangelos Kiskinis
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Abstract
The present disclosure relates generally to compositions and methods for inhibiting dipeptide repeat protein (DPR)-ribosomal RNA (rRNA) interaction. In particular, the present technology relates to administering a therapeutically effective amount of one or more compositions that inhibit DPR-rRNA interaction to a subject diagnosed with, or at risk for DPR-associated pathologies, e.g., amyotrophic lateral sclerosis or frontotemporal dementia.
Claims23
A single-stranded RNA (ssRNA) construct comprising a sequence that is identical to the sequence of a fragment of a ribosomal RNA (rRNA) subunit selected from the group consisting of the 28S, 18S, and 5.8S subunits, or a fragment of the 5′ETS or ITS1 region of the 47S rRNA precursor, wherein the ssRNA construct comprises one or more modified nucleotides.
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A method for treating or preventing amyotrophic lateral sclerosis or frontotemporal dementia in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of an ssRNA construct that inhibits dipeptide repeat (DPR) protein-ribosomal RNA (rRNA) interaction, wherein the ssRNA construct comprises a sequence that is identical to the sequence of a fragment of a ribosomal RNA (rRNA) or a rRNA precursor.
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Dependencies are inferred from the claim text.