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. 27, 2022
Filed:
Mar. 14, 2022
The Board of Trustees of the Leland Stanford Junior University, Stanford, CA (US);
Matthew H. Porteus, Stanford, CA (US);
Ayal Hendel, Stanford, CA (US);
Joe Clark, Stanford, CA (US);
Rasmus O. Bak, Stanford, CA (US);
Daniel E. Ryan, Santa Clara, CA (US);
Douglas J. Dellinger, Santa Clara, CA (US);
Robert Kaiser, Santa Clara, CA (US);
Joel Myerson, Santa Clara, CA (US);
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY, Stanford, CA (US);
AGILENT TECHNOLOGIES, INC., Santa Clara, CA (US);
Abstract
Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.