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US 2025/0059543 A1Published application
Methods and compositions for targeted trans-splicing
AssigneeAmber Bio Inc.
InventorsJacob BORRAJO, Basem AL-SHAYEB
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Abstract
The present disclosure provides compositions and methods of use thereof for targeting trans-splicing of a pre-mRNA in a cell.
Claims31
1-117. (canceled)
A method for trans-splicing a pre-mRNA target sequence comprising:
- contacting a cell comprising the pre-mRNA target sequence with one or more trans-splicing RNA molecules comprising:
- (i) at least one sequence comprising a non-coding RNA (ncRNA), wherein the ncRNA is a small nucleolar RNA (snoRNA) sequence of about 7 to about 300 nucleotides in length;
- (ii) at least one splice acceptor site or splice donor site;
- and
- (iii) at least one exonic sequence;
- wherein at least a portion of the one or more trans-splicing RNA molecules recognizes one or more pre-mRNA target sequences;
- and
- splicing the at least one exonic sequence into the pre-mRNA target sequence.
5 more elements
Dependencies are inferred from the claim text.