Minimal RNA Targeting CRISPR Cas Systems

Patent Status

Patent Pending

Brief Description

UC Berkeley researchers have indentified and characterized a novel CRISPR Cas13 subtype that exhibits unique and advantageous features for transcriptome editing applications. At approximately half the size of the smallest known Cas13 subtype, this novel subtype is the smallest CRISPR Cas effector identified to date. The compactness of this novel Cas13 subtype facilitates its delivery into a wide array of cell types using various delivery mechanisms, significantly enhancing its utility in genomic research and therapeutic applications. The novel Cas13 subtype retains the hallmark programmable RNA-targeting capability of the Cas13 family, enabling precise and efficient editing of RNA sequences. This feature is particularly valuable in the context of transcriptome engineering, where specific alterations to RNA molecules can modulate gene expression, correct genetic errors, or modulate the function of non-coding RNAs. The discovery of this compact Cas13 subtype opens new avenues for transcriptome editing, offering potential applications in functional genomics, gene therapy, and the development of novel therapeutic strategies targeting RNA. Its ease of delivery and potent RNA-editing capabilities position this novel Cas13 subtype as a valuable tool for both basic research and clinical applications in the field of genetic engineering and precision medicine.

Suggested uses

• Transcriptome editing
• Gene therapy
• Therapeutic strategies targeting RNA

Advantages

• The small size of this novel subtype does not compromise its efficiency or specificity in targeting RNA, which is a significant advancement over existing Cas13 variants that are larger and more challenging to deliver into target cells.