Compositions comprising chemically modified guide RNAs for CRISPR/Cas-mediated editing of HBB

US11851652B2 · kind B2 · utility

Assignees

• The Board of Trustees of the Leland Stanford Junior · US
• UNIVERSITY and AGILENT TECHNOLOGIES, INC. · US

Inventors

• Matthew H. Porteus · Stanford, US
• Ayal Hendel · Stanford, US
• Joe Clark · Stanford, US
• Rasmus O. Bak · Stanford, US
• Daniel E. Ryan · San Francisco, US
• Douglas J. Dellinger · Boulder, US
• Robert Kaiser · Bothell, US
• Joel Myerson · Berkeley, US

Key dates

• Filing date: Mar 14, 2022
• Grant date: Dec 26, 2023
• Expiry date: Mar 14, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/51
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.