Methods of Modulating Expression of Target Nucleic Acid Sequences in A Cell
AssigneePresident and Fellows of Harvard College
InventorsSuhani D. Vora, Alejandro Chavez, Jing Cheng, Benjamin W. Pruitt
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Abstract
The present disclosure provides methods and compositions of modulating expression of a target nucleic acid sequence in a cell. The method comprises introducing into the cell a nucleic acid sequence encoding a Cas9 fusion protein and a guide RNA, wherein the Cas9 fusion protein and the guide RNA are expressed and co-localize at a target site and modulate the expression of the target nucleic acid sequence.
Claims53
A method of modulating expression of a target nucleic acid sequence in a cell comprising:
- introducing into the cell a nucleic acid sequence encoding a Cas9 fusion protein and a guide RNA, wherein the Cas9 fusion protein comprises Cas9 fused with at least one modified activation domain of VP64, p65 or RTA, or Cas9 fused with a combination of at least two modified activation domains of VP64, p65 and RTA, wherein the Cas9 fusion protein and the guide RNA are expressed and co-localize at a target site and modulate the expression of the target nucleic acid sequence.
A nucleic acid construct comprises nucleic acid sequences encoding a Cas9 fusion protein and a guide RNA.
A method of treating a disease of a subject comprising
- administering a therapeutically effective amount of a recombinant AAV(rAAV) in the subject in need thereof.
Dependencies are inferred from the claim text.