Back to results
US 11,530,425 B2Granted
Systems, methods, and compositions for correction of frameshift mutations
AssigneeMassachusetts Institute of Technology
InventorsTimothy Lu, Shota Nakade
Life of this patent
Estimating the term…
Abstract
The disclosure provides systems, methods, and compositions for a target specific nuclease and a blunting enzyme to correct frameshift mutations for genome editing and treatment of diseases. In some embodiments, the target specific nuclease and the blunting enzyme are combined with a guide RNA and/or a microhomology-mediated end joining (MMEJ) inhibitor.
Claims17
A composition comprising:
- (a) a target specific nuclease, wherein a target comprises a double-stranded DNA (dsDNA);
- and
- (b) a double strand break (DSB)-end blunting enzyme;
- wherein the target specific nuclease is selected from the group consisting of Cas12a, LbCas12a, FnCas12a, AsCas12a, Cas9, SpCas9, SaCas9, LZ3Cas9, Casccφ, and the double combinations of Cas9 nickase, zinc finger nuclease (ZFN), and TAL Effector Nuclease (TALEN),
- wherein the composition further comprises an inhibitor of the microhomology-mediated end joining (MMEJ) pathway, and
- wherein the MMEJ pathway inhibitor is a CtIP inhibitor selected from KLHL15 and PIN1.
3 more elements
Dependencies are inferred from the claim text.