Compositions and Methods for Modifying Dystrophin Genes
AssigneeThe Regents of the University of California
InventorsMelissa J. Spencer, Courtney S. Young, April D. Pyle, Michael Reza Emami
Life of this patent
Estimating the term…
Abstract
Disclosed herein are guide sequences for modifying a dystrophin gene using CRISPR technology. Specifically, the disclosure provides a method of modifying a dystrophin gene in a cell or a subject, which comprises introducing into the cell or subject (a) a Cas protein or a nucleotide sequence encoding the Cas protein; and a single guide RNA (gRNA), or a first gRNA and a second gRNA, wherein the Cas protein is a type II CRISPR/Cas endonuclease. Further disclosed are gRNA nucleic acid sequences.
Claims14
A nucleic acid molecule which comprises or consists of a sequence having 100% sequence identity to at least 17, at least 18, or at least 19 nucleotides of a sequence selected from the group consisting of SEQ ID NOs:
- 1-43 and 87-118, preferably selected from the group consisting of SEQ ID NOs:
- 1-41 and 87-118.
A guide RNA comprising a guide sequence having 100% sequence identity to at least 17, at least 18, or at least 19 nucleotides of a sequence selected from the group consisting of SEQ ID NOs:
- 1-41.
Dependencies are inferred from the claim text.