Methods and compositions for treatment of a genetic condition
Methods and compositions comprising a fusion protein comprising a Cas protein and a functional domain in association with a single guide RNA targeted to an endogenous gene for genetic alteration of cells are provided.
1. A fusion protein comprising a Cas protein and a functional domain in association with a single-guide RNA comprising a sequence selected from the group consisting of SEQ ID NOs:149-158 and 167-215.
2. The fusion protein of claim 1 , wherein the functional domain is a transcriptional activation domain, a transcriptional repression domain or a nuclease domain.
3. The fusion protein of claim 2 , wherein the nuclease domain is a FokI endonuclease domain.
4. The fusion protein of claim 3 , wherein the FokI endonuclease domain comprises one or more mutations in the dimerization domain.
5. A dimer comprising first and second fusion proteins according to claim 3 .
6. The dimer of claim 5 , wherein the first fusion protein comprises a FokI endonuclease domain comprising one or more mutations in the catalytic domain.
7. A pharmaceutical composition comprising a fusion protein according to claim 1 .