CRISPR ENZYMES AND SYSTEMS

Smart overview of the Invention

The patent application describes innovative systems, methods, and compositions for targeting nucleic acids using engineered CRISPR systems. It introduces a non-naturally occurring DNA-targeting CRISPR effector protein, alongside at least one targeting nucleic acid component, such as a guide RNA. These novel systems are designed for precise genome editing and manipulation, which can significantly enhance genetic research and applications in biotechnology and medicine.

Background and Need

With the rapid advancements in genome sequencing techniques, there is an increasing demand for precise genome-targeting technologies. Existing methods like designer zinc fingers and TALEs have limitations, prompting the need for new technologies that are scalable, cost-effective, and capable of targeting multiple genomic positions. The CRISPR-Cas systems, known for their diversity and adaptability, offer promising solutions. This invention leverages these systems to develop powerful genome engineering tools.

Technical Specifications

The invention outlines a method for modifying sequences at a target locus using a Type V CRISPR-Cas loci effector protein. This protein forms a complex with nucleic acid components to induce modifications at the target site. The application emphasizes the use of Cpf1 effector proteins and describes their role in introducing strand breaks in DNA sequences. The CRISPR RNA (crRNA) or guide RNA plays a crucial role in directing these modifications.

Innovative Features

This invention highlights the engineering of Cpf1 effector protein complexes with optimized nucleic acid components. These components may include guide sequences linked to direct repeat sequences with stem loops or optimized secondary structures. The application also explores the incorporation of protein-binding RNA aptamers within these structures, enhancing their functionality by binding to specific bacteriophage coat proteins like MS2.

Applications and Implications

The described CRISPR systems have broad applications in genomic and epigenomic targeting, enabling direct detection, analysis, and manipulation of specific genetic sites. By expanding the toolkit available for genetic engineering, this invention holds potential for transformative impacts on research methodologies and practical applications in various fields such as synthetic biology and medical therapeutics.