Genome-editing methods like CRISPR-Cas9 are being studied to help treat cancer and sickle cell disease. St. Jude scientists are creating tools to better understand how the system works.
Genome editing must be precise to be used as therapy. However, factors that affect off-target activity are largely unknown.
Variation among genomes can also affect editing activity. It is crucial to have methods that can be applied to individual genomes to understand safety.
“CHANGE-seq is the first truly scalable method for studying the unintended activity of genome editors,” said Shengdar Tsai, PhD, of Hematology. “We can now rapidly pick the best and safest genome editors and targets for therapeutic editing.”
Nature Biotechnology published a report on this work.