Delivery, use and therapeutic applications of the CRISPR-cas systems and compositions for modeling mutations in leukocytes

US11197467B2 · kind B2 · utility

Assignees

• The Broad Institute, Inc. · US
• Massachusetts Institute of Technology · US
• The General Hospital Corporation · US

Inventors

• Aviv Regev · Cambridge, US
• Oren Parnas · Jerusalem, IL
• Marko Jovanovic · Berlin, DE
• Nir Hacohen · Cambridge, US
• Thomas Eisenhaure · North Reading, US

Key dates

• Filing date: Mar 24, 2017
• Grant date: Dec 14, 2021
• Expiry date: Mar 24, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The invention involves a method for modulating leukocyte activity, comprising delivering to a leukocyte a vector containing nucleic acid molecule(s), whereby the leukocyte contains Cas9 and the vector expresses one or more RNAs to guide the Cas9 to introduce mutations in one or more target genetic loci in the leukocyte, thereby modulating expression of one or more genes expressed in the leukocyte. The invention also involves identifying genes associated with leukocyte responses and experimental modeling of aberrant leukocyte activation and diseases associated with leukocytes by introducing mutations into leukocytes. The invention comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate leukocyte associated diseases.