RNA-guided gene editing and gene regulation
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.
Claims10
A DNA targeting system for deleting exon 51 of a dystrophin gene comprising Cas9 and at least one guide RNA (gRNA), wherein the at least one gRNA comprises a polynucleotide sequence corresponding to SEQ ID NO:
- 65, SEQ ID NO:
- 679, or SEQ ID NO:
- 69.
Dependencies are inferred from the claim text.