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US 2025/0154533 A1Published application
Engineered retrons and methods of use
AssigneeReNAgade Therapeutics Management Inc.
InventorsAlim Ladha, Vladimir Presnyak, Inna Shcherbakova, Brian Goodman, Mario Rodriguez Mestre, Devin Scott Quinlan, Muthusamy Jayaraman, Stephen Scully
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Abstract
Disclosed are engineered retrons and methods of use such as to modify the genome of a host (e.g., mammalian) cell by delivering the engineered retron or the encoded ncRNA in vitro or in vivo to the host (e.g., mammalian) cell.
Claims28
1-30. (canceled)
A gene editing system comprising one or more delivery vehicles, wherein:
- the delivery vehicle(s) comprise RNA cargo,
- said RNA cargo comprises (a) at least one mRNA molecule encoding (i) a nucleic acid programmable nuclease and (ii) a retron reverse transcriptase, (b) an engineered retron ncRNA, and (c) guide RNA for the nucleic acid programmable nuclease;
- wherein the engineered retron ncRNA comprises an HDR nucleotide sequence substituted into a retron ncRNA;
- wherein the retron ncRNA nucleotide sequence has about 85% to 98% sequence identity to any one of SEQ ID NO:14826, SEQ ID NO:
- 17305, or SEQ ID NO:18409;
- wherein the HDR nucleotide sequence is substituted at a hairpin loop of the retron ncRNA;
- wherein the retron reverse transcriptase and the retron ncRNA are from the same phylogenetic clade;
- each delivery vehicle contains (a)(i) and/or (a)(ii) and/or (b) and/or (c),
- whereby one delivery vehicle or more than one delivery vehicle delivers (a)(i), (a)(ii), (b), and (c).
6 more elements
Dependencies are inferred from the claim text.