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Meta‐analysis of Gene-edited Cell Persistence

Gene editing of hematopoietic cells represents a promising, yet still experimental, strategy to correct genetic disorders. A new STEM CELLS Translational Medicine article from the lab of David S. Allan (Ottawa Hospital Research Institute, ON, Canada) reports on a systematic review of the literature and the identification of fifteen animal studies that transplanted blood‐forming stem cells edited with CRISPR/Cas9 technology. While edited cells engrafted similarly to unedited cells, a meta‐analysis revealed that edited cells declined over time after transplantation, thereby highlighting the need for new approaches to target hematopoietic stem cells. Furthermore, Maganti et al. suggest that future studies should reduce potential sources of bias by blinding outcome assessors and randomizing animals to accelerate translation to clinical studies.