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US 12,686,869 B1Granted
Methods and compositions for trans-splicing utilizing trifunctional elements
AssigneeAmber Bio Inc.
InventorsJacob Borrajo, Brigit Riley, Kamyab Javanmardi, Patricia Biezonski, Samuel Demario
Life of this patent
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Abstract
The present disclosure provides a trans-splicing molecule that has a trifunctional element suitable for targeted trans-splicing of an RNA or pre-mRNA molecule.
Claims27
A trans-splicing molecule comprising a trifunctional element suitable for targeted trans-splicing of an RNA or pre-mRNA molecule, comprising:
- (i) one or more stabilizing structural elements having at least 95% sequence identity to SEQ ID NO:
- 216 or 274;
- (ii) one or more cleavage elements having at least 95% sequence identity to SEQ ID NO:
- 217;
- and
- (iii) a termination sequence having at least 95% sequence identity to SEQ ID NO:
- 218.
4 more elements
A trans-splicing molecule comprising a trifunctional element suitable for targeted trans-splicing of an RNA or pre-mRNA molecule, comprising:
- (i) one or more stabilizing structural elements,
- (ii) one or more cleavage elements, and
- (iii) a termination sequence,
- wherein the trans-splicing molecule has at least 99% identity to one of SEQ ID NOs:
- 255-258.
2 more elements
Dependencies are inferred from the claim text.