Is a
Patent attributes
Patent Jurisdiction
Patent Number
Patent Inventor Names
Douglas J. Dellinger0
Robert Kaiser0
Rasmus O. Bak0
Matthew H. Porteus0
Joel Myerson0
Joe Clark0
Ayal Hendel0
Daniel E. Ryan0
Date of Patent
April 19, 2022
0Patent Application Number
157240730
Date Filed
October 3, 2017
0Patent Citations
Patent Citations Received
Patent Primary Examiner
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.
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