TRANSLATION ENHANCING NUCLEIC ACID COMPOUNDS: ASO COUPLED TRANSLATION – UPREGULATION 1 (ACT-UP1) AND USES THEREOf
AssigneeArnatar Therapeutics, Inc
InventorsXuehai Liang, Lingdi Zhang
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Abstract
Disclosed herein are methods and compounds for enhancing gene expression by ACT-UP1 compounds. Such methods and compounds are useful for increasing expression of certain genes, many of which are associated with a variety of diseases and disorders.
Claims34
An antisense oligonucleotide (ASO) Coupled Translation-Upregulation 1 (ACT-UP1) compound, wherein the ACT-UP1 compound comprises an ASO component joined to a protein recruiting sequence (PRS) component, wherein the ASO component is capable of hybridizing to a target mRNA, wherein the PRS is capable of recruiting translation related proteins, and wherein the ACT-UP1 compound is capable of enhancing protein expression.
A pharmaceutical composition comprising the ACT-UP1 compound of any preceding claim, alone or in combination with a pharmaceutically acceptable carrier and/or excipient.
Dependencies are inferred from the claim text.