CAS9 fusion proteins, RNA-guided gene editing, gene regulation compositions, and methods comprising the same
AssigneeDuke University
InventorsCharles A. Gersbach, Isaac B. Hilton, Pablo Perez-Pinera, Ami M. Kabadi, Pratiksha I. Thakore, David G. Ousterout, Joshua B. Black
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Abstract
Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.
Claims15
A fusion protein comprising two heterologous polypeptide domains, wherein the first polypeptide domain comprises a Clustered Regularly Interspaced Short Palindromic Repeats associated (Cas) protein comprising a Cas9 derived from Streptococcus pyogenes and the mutations D10A and H840A to inactivate the nuclease activity of Cas9, and wherein the second polypeptide domain comprises a p300 histone acetyltransferase effector domain comprising a wild-type human p300 protein or the core lysine-acetyltransferase domain of the human p300 protein.
Dependencies are inferred from the claim text.