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US 2026/0218187 A1Published application
Materials and methods for modulating expression
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsKathrin Meyer, Shibi Likhite, Samantha Lynn Powers
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Abstract
The present disclosure relates to methods of modulating transcription of target genes using transcription modulators termed RNA-based transcription modulators or “RPMs”. The RPMs attract transcription regulators to the genes.
Claims18
An RNA-based promoter modulator (RPM) comprising:
- (a) a regulatory element tail,
- (b) a binding sequence, and
- (c) an Sm OPT consensus sequence and hairpin.
An SMN2 RPM comprising:
- (a) Regulatory Element A Tail encoded by
- (SEQ ID NO:
- 1)
- ACCCTTCTCCGGCCGCTGAC
- or
- Regulatory Element B Tail encoded by
- (SEQ ID NO:
- 2)
- TCCGCTCTGGGGCGCGCACACCCTCGCCCGCACTTCTCCCCTCGTCCGG
- CACGGACTGACCGCACACGAATAACACATCCGACCAGAGCT,
- and
- (b) Binding Sequence 1 encoded by
- (SEQ ID NO:
- 3)
- AGACGGGGTTTCGGCATGTT,
- Binding Sequence 2 encoded by
- (SEQ ID NO:
- 4)
- ATTGTGTAGGCTGGTCTGA,
- or
- Binding Sequence 3 encoded by
- (SEQ ID NO:
- 5)
- GGGGCAGGAAGGAAGGCAGA.
21 more elements
A TCF4 RPM comprising:
- (a) Regulatory Element A Tail encoded by
- (SEQ ID NO:
- 1)
- ACCCTTCTCCGGCCGCTGAC
- or
- Regulatory Element B Tail encoded by
- (SEQ ID NO:
- 2)
- TCCGCTCTGGGGCGCGCACACCCTCGCCCGCACTTCTCCCCTCGTCCGG
- CACGGACTGACCGCACACGAATAACACATCCGACCAGAGCT,
- and
- (b) Binding Sequence 1 encoded by
- (SEQ ID NO:
- 6)
- GTGGTAAACAGAGCGCCTAG.
12 more elements
Dependencies are inferred from the claim text.