Methods and compositions for regulating gene expression
AssigneeThe University of Chicago
InventorsChuan HE, Hui-Lung SUN, Yan-Ming CHEN
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Abstract
Aspects of the present disclosure are directed to at least methods and compositions for site-specific targeting or RNA and/or pseudouridine installation in RNA, such as mRNA. Provided targeting and/or pseudouridine installation can increase stability of RNA, increase mRNA translation, and/or fine-tune protein expression. Also disclosed herein are compositions, methods, and kits suitable for therapeutic application of site-specific targeting of RNA and/or pseudouridine installation in RNA.
Claims75
A composition comprising, a pseudouridine synthase protein linked to a targeting protein.
A composition comprising one or more targeting RNA molecules that target a messenger RNA (mRNA) molecule associated with a target gene.
A method of modulating translation of a target mRNA comprising, contacting one or more site-specific rare codons in the target mRNA with a) one or more site-specific targeting elements, and/or b) one or more of a pseudouridine synthase protein and/or one or more box H/ACA small nucleolar ribonucleoprotein (H/ACA snoRNP) complex components.
The method of claim 38 or 39, wherein the Cas protein comprises a dCas13d protein.
Dependencies are inferred from the claim text.