Translation enhancing nucleic acid compounds: ASO coupled translation-upregulation 1 (ACT-UP1) and uses thereof
AssigneeArnatar Therapeutics, Inc
InventorsXuehai Liang, Lingdi Zhang
Life of this patent
Estimating the term…
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.
Claims21
A compound comprising an oligonucleotide for enhancing expression of JAG1 in a cell or salt thereof, wherein the oligonucleotide comprises at least a 20 nucleobase portion of the sequence of SEQ ID NO:
- 111, or a sequence thereof which differs up to 2 positions of SEQ ID NO:
- 111, wherein the compound further comprises a conjugate.
A compound comprising an oligonucleotide for enhancing expression of JAG1 in a cell or salt thereof, wherein the oligonucleotide comprises at least a 20 nucleobase portion of the sequence of SEQ ID NO:
- 111, or a sequence thereof which differs up to 2 positions of SEQ ID NO:
- 111, wherein the compound comprises at least one chemical modification.
A compound comprising an oligonucleotide for enhancing expression of JAG1 in a cell or salt thereof, wherein the oligonucleotide comprises at least a 20 nucleobase portion of the sequence of SEQ ID NO:
- 111, or a sequence thereof which differs up to 2 positions of SEQ ID NO:
- 111, wherein the compound is fully chemically modified.
A compound comprising an oligonucleotide for enhancing expression of JAG1 in a cell or salt thereof, wherein the oligonucleotide comprises at least a 20 nucleobase portion of the sequence of SEQ ID NO:
- 111, or a sequence thereof which differs up to 2 positions of SEQ ID NO:
- 111, wherein the compound comprises at least one modified internucleoside linkage.
Dependencies are inferred from the claim text.