The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Dec. 24, 2024
Filed:
Feb. 04, 2022
Christiana Care Gene Editing Institute, Inc., Wilmington, DE (US);
Eric B. Kmiec, Middletown, DE (US);
Byung-Chun Yoo, Newark, DE (US);
Pawel Bialk, Wilmington, DE (US);
Abstract
This disclosure relates to methods of and compositions for reducing expression or activity of a variant gene comprising at least one mutation as compared its wild-type gene, comprising introducing into a cell comprising the variant gene one or more DNA sequences encoding two or more gRNAs that are complementary to two or more target sequences in the variant gene, wherein at least one of the gRNAs hybridizes to a target sequence comprising a PAM site in the variant gene that results from a mutation to the variant gene creating the PAM site that does not exist in the wild-type gene or is operably linked to a mutated portion of the wild-type gene, at least one of the gRNAs hybridizes to a target sequence comprising a PAM site in an intron of the variant gene downstream or upstream from the PAM site, and a nucleic acid sequence encoding a CRISPR-associated endonuclease; wherein a CRISPR-associated endonuclease cleaves the variant gene at the target sequences; and expression or activity of the variant gene is reduced in the cell relative to a cell in which the one or more DNA sequences encoding the two or more gRNAs and the nucleic acid sequence encoding the CRISPR-associated endonuclease are not introduced.