Periodic Reporting for period 1 - CRISPR-SELECT (Harnessing a novel CRISPR nuclease for programmable counterselection in human cells)
Periodo di rendicontazione: 2024-04-01 al 2025-09-30
CRISPR technologies and their creation of defined DNA edits in virtually any organism are revolutionizing medicine, agriculture, basic research, and biotechnology. However, editing is often inefficient, requiring additional and often laborious screening steps. My group’s recent work, which stemmed from my ERC Consolidator Award, offers a unique opportunity: achieving the first means to select for a desired gene edit in eukaryotic cells. Such a capability could relieve this pain point, becoming a highly desirable approach across many applications of genome editing. Through this project, we will achieve a proof-of-concept demonstration and lay the groundwork for commercialization and widespread adoption.
The project achieved the proof-of-principle demonstration of enriching gene edits in human cells through two modalities: indel formation and prime editing. The resulting work is currently in submission as a revised manuscript.
Further IP was secured through a variant of the Cas12a2 nucleases called Cas12a3. Future implementation will require follow-on demonstrations, development of guide rules, and expansion to other cell lines and organisms, such as plants.