What does “optimized” preserve in primary hematopoietic cells?
by Pavel Ilyan
Optimization of CRISPR/Cas9 editing in primary human hematopoietic cells should not collapse efficiency and specificity into one endpoint. Which assay quantifies the complete intended-locus product spectrum, including indels and larger structural outcomes? Are nearby sequence changes measured separately from genome-wide off-target events, and are these outcomes resolved across relevant cell subsets rather than only in pooled cells?
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