Researchers identified efficient CRISPR guide pairs for removing the C9orf72 repeat expansion in patient stem cells
Original source
Arrayed dual-gRNA CRISPR screening platform for C9orf72 repeat expansion excision in patient iPSCs. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Population inferred from the title and abstract by Compass.
Related topics
A study screened 120 dual-guide CRISPR-Cas9 combinations in patient-derived induced pluripotent stem cells (iPSCs) carrying the C9orf72 repeat expansion. Excision of both copies of the region was more efficient than targeting only the expanded copy. The researchers detected one off-target change among the four most efficient guides tested.
Why this matters
The findings may help guide development of gene-editing approaches for C9orf72-related frontotemporal dementia and amyotrophic lateral sclerosis (ALS). This was work in patient iPSCs, not in people, so it does not currently change treatment for anyone with ALS or frontotemporal dementia.
Limitations and context
The study was conducted in patient-derived iPSCs rather than animals or people. Its safety assessment was limited to the tests and guides described in the paper, and further work would be needed to establish whether the approach works safely and effectively in relevant cells, tissues, or patients.