Crispr-cas genome engineering via a modular aav delivery system
AssigneeThe Regents of the University of California
InventorsPrashant Mali, Dhruva Katrekar, Ana Collado
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Abstract
The present disclosure relates to a novel delivery system with unique modular CRIPSR-Cas9 architecture that allows better delivery, specificity and selectivity of gene editing. It represents significant improvement over previously described split-Cas9 systems. The modular architecture is “regulatable”. Additional aspects relate to systems that can be both spatially and temporally controlled, resulting in the potential for inducible editing. Further aspects related to a modified viral capsid allowing conjugation to homing agents.
Claims14
A recombinant expression system for CRISPR-based genome or epigenome editing comprising:
- (a) a first expression vector comprising (i) a polynucleotide encoding C-intein, (ii) a polynucleotide encoding C-Cas9, and (iii) a promoter sequence for the first vector;
- and
- (b) a second expression vector comprising (i) a polynucleotide encoding N-Cas9, (ii) a polynucleotide encoding N-intein, and (iii) a promoter sequence for the second vector, wherein optionally, both the first and second expression vectors are adeno-associated virus (AAV) or lentivirus vectors, and
- wherein co-expression of the first and second expression vectors results in the expression of a whole Cas9 protein.
Dependencies are inferred from the claim text.