Methods for identifying and targeting non-coding rna scaffolds
AssigneeTranslate Bio MA, Inc.
InventorsJames BARSOUM, Caroline WOO
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Abstract
Aspects of the disclosure provide steric-blocking oligonucleotide-based methods of modulating expression of target genes, e.g., by targeting non-coding RNA scaffolds.
Claims47
A method of producing a steric-blocking oligonucleotide, the method comprising:
- determining that a non-coding RNA scaffold has a first interaction region that interacts with a repressor of a target gene and a second interaction region that interacts with an activator of the target gene;
- and
- producing a steric-blocking oligonucleotide having a region of complementarity that is complementary with the first interaction region or the second interaction region.
A method of preparing a steric-blocking oligonucleotide, the method comprising:
- determining that a non-coding RNA scaffold interacts with an activator of the target gene and a repressor of the target gene;
- identifying an interaction region of the non-coding RNA that interacts with either the activator or the repressor, but not both;
- and
- preparing a steric-blocking oligonucleotide having a region of complementarity that is complementary with the interaction region.
A method of modulating expression of a target gene in a cell, the method comprising:
- delivering to the cell an effective amount of a steric-blocking oligonucleotide, wherein the cell expresses a non-coding RNA scaffold, wherein prior to delivering the steric-blocking oligonucleotide it has been determined that the non-coding RNA scaffold has a first interaction region that interacts with a repressor of the target gene and a second interaction region that interacts with an activator of the target gene, and wherein the steric-blocking oligonucleotide has a region of complementarity that is complementary with the first interaction region or the second interaction region.
A method of modulating expression of a target gene in a cell, wherein it has been determined that a non-coding RNA interacts with both an activator of the target gene and a repressor of a target gene, the method comprising:
- delivering to the cell a steric-blocking oligonucleotide having a region of complementarity that is complementary with a region of the non-coding RNA that interacts with either the activator or the repressor, but not both.
A method of increasing expression of a target gene in a cell, the method comprising:
- delivering to the cell an effective amount of a steric-blocking oligonucleotide, wherein the cell expresses a non-coding RNA, wherein prior to delivering the steric-blocking oligonucleotide it has been determined that the non-coding RNA scaffold has a first interaction region that interacts with a repressor of the target gene and a second interaction region that interacts with an activator of the target gene, wherein the steric-blocking oligonucleotide has a region of complementarity that is complementary with the first interaction region.
A method of increasing expression of a target gene in a cell, the method comprising:
- delivering to the cell a steric-blocking oligonucleotide having a region of complementarity that is complementary with a region of the non-coding RNA scaffold that interacts with a repressor of the target gene, wherein displacement of the repressor from the non-coding RNA, without displacement an activator of the target gene that also interacts with the non-coding RNA scaffold, indicates effectiveness of the steric-blocking oligonucleotide.
Dependencies are inferred from the claim text.