Yifan Zhou

TL;DR: A rhesus macaque model of human ARCH utilizing CRISPR/Cas9 technology and RNA-seq indicated that TET2-disrupted myeloid colony-forming units (CFUs) and mature cells exhibited a distinct hyperinflammatory gene expression profile, suggesting that other intrinsic and/or extrinsic factors can regulate the pace of Tet2 clonal expansion.

TL;DR: A rhesus macaque model is utilized to ask whether CIRCLE-Seq (CS), an in vitro off- target prediction method, more accurately identifies off-targets compared to in silico prediction (ISP) based solely on genomic sequence comparisons, and whether a combination of careful gRNA design, followed by screening for predicted off-target sites in target cells by multiple methods may be required for optimizing safety of clinical development.

TL;DR: Overall, in this nonhuman primate model with great relevance to human hematopoiesis, this work documented continuous production of erythroid cells from multipotent, non-biased hematoietic stem cell clones at steady-state or under stress.

TL;DR: This work adapted CRISPR/Cas9 editing of T cells for precise genome editing at a site of interest and has accelerated human disease modeling and the development of corrective gene therapies for various genetic disorders.