Fanzors are RNA-Guided Nucleases Encoded in Eukaryotic Genomes

This invention introduces compositions and methods for targeting polynucleotides with eukaryotic RNA-guided nucleases, particularly Fanzors which are programmable RNA-guided DNA endonucleases, useful for genome editing in human cells and other organisms. Compositions include Fanzor polypeptides with RuvC domains and fRNA molecules directing them to target specific sequences. This technology enables precise targeting of specific polynucleotide sequences, highlighting the biotechnological potential of eukaryotic RNA-guided nucleases and potentially revolutionizing gene editing and therapeutic applications.

Researchers

Omar Abudayyeh / Jonathan Gootenberg / Justin Lim / Kaiyi Jiang

Departments: McGovern Institute for Brain Research, Biological Engineering
Technology Areas: Biotechnology: DNA & RNA Editing, Synthetic Biology / Therapeutics: Nucleic Acids
Impact Areas: Healthy Living

  • fanzors are rna-guided nucleases encoded in eukaryotic genomes
    United States of America | Pending
  • fanzors are rna-guided nucleases encoded in eukaryotic genomes
    Patent Cooperation Treaty | Pending

Technology 

The Fanzor polypeptide consists of two main parts: a RuvC domain and an fRNA molecule. The fRNA molecule acts as a guide, directing the Fanzor polypeptide to a specific target sequence in the genetic material. The RuvC domain within the Fanzor polypeptide plays a crucial role to cleave/modify the target sequence. As a whole, the Fanzor protein acts as an enzyme to allow precise manipulation of genetic material. 

Problem Addressed 

Challenges in gene editing lie in accurately editing specific DNA or RNA sequences without affecting other parts of the genome. Furthermore, existing technologies tend to lack precision or efficiency, creating a need for safe and effective gene editing tools for research and medicine. Fanzors address these problems by using RNA-guided endonucleases to precisely target specific genetic sequences. This innovation has the potential to revolutionize gene editing and therapeutic applications while minimizing risks associated with unintended mutations. 

Advantages

  • Precise targeting of DNA or RNA sequences 
  • Improved safety and efficiency compared to existing methods 
  • Versatility for various research, clinical, and therapeutic applications 

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