Crispr enzymes and systems

US11352647B2 · kind B2 · utility

Assignees

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

Inventors

• Feng Zhang · Cambridge, US
• David A. Scott · Cambridge, US
• Winston X. Yan · Brookline, US
• Sourav Roy Choudhury · Newton, US
• Matthias Heidenreich · Cambridge, US

Key dates

• Filing date: Aug 17, 2017
• Grant date: Jun 7, 2022
• Expiry date: Aug 17, 2037

Classification

• Technology area (CPC G): Physics
• CPC primary: G16B30/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity. Thus, in another aspect certain embodiments disclosed herein are directed to delivery vectors, constructs, and methods of delivering larger genes for systemic delivery.