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US 2026/0308553 A1Published application
Cas-embedded Cytidine Deaminase Ribonucleoprotein Complexes having Improved Base Editing Specificity and Efficiency
AssigneeUniversity of Massachusetts
InventorsJeong Min Lee, Nese Kurt Yilmaz, Celia A. Schiffer
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Abstract
Some aspects of the disclosure provide Cas-embedded cytosine base editors with expanded or shifted editing windows, allowing for editing of cytosines (Cs) at otherwise inaccessible positions. Methods of treating a disease or disorder using the Cas-embedded cytosine base editors are also provided.
Claims21
A fusion protein comprising, in an N to C direction:
- (i) an N-terminal domain of a Cas9 nickase (N-nCas9);
- (ii) a first linker sequence;
- (iii) a cytidine deaminase domain comprising SEQ ID NO:2;
- (iv) a second linker sequence;
- (v) a C-terminal domain of the Cas9 nickase (C-nCas9);
- and
- (vi) a uracil glycosylase inhibitor sequence (UGI).
4 more elements
21-37. (canceled)
Dependencies are inferred from the claim text.