Genomic Insertion of Large Cargo with Engineered Retro Elements

Genome editing tools for use in systems designed to deliver large genetic elements are disclosed herein. A genome editing system is described, which includes i) an R2 element enzyme or other non-LTR site specific retrotransposon element and ii) a payload RNA, wherein the payload RNA comprises an insertion region and optionally one or more of a 5' homology region, a 3' homology region, and a protein binding element, wherein the insertion region comprises a template for a small or large nucleic acid insertion into the genome, and wherein the R2 element enzyme or other non-LTR site specific retrotransposon element comprises a targeting domain, a reverse transcriptase domain, and a nickase domain. Also disclosed are cells edited using such a genome editing system, methods for editing a genome, and compositions comprising cells edited with this genomic editing system.

Researchers

Omar Abudayyeh / Justin Lim / Lukas Villiger / Jonathan S. Gootenberg

Departments: McGovern Institute for Brain Research
Technology Areas: Drug Discovery and Research Tools: Genomics & Proteomics / Therapeutics: Cell Based Therapy

  • genomic editing with site-specific retrotransposons
    United States of America | Published application
  • genomic editing with site-specific retrotransposons
    European Patent Convention | Published application

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