Mark A. DeWitt

The paper develops scOUT-seq to profile DNA repair outcomes in single cells and finds that repair outcomes vary significantly across cell types and subtypes.

This paper develops NanoCas, a compact CRISPR nuclease, and demonstrates its efficacy in editing non-liver tissues in non-human primates.

This study uses CRISPR base editing to identify novel mutations in the HBG promoter that increase fetal hemoglobin expression without harmful effects.

The paper identifies ATF4 as a key regulator of fetal globin re-expression in response to reduced β-globin, providing mechanistic insight into stress-induced globin compensation.

This study uses CRISPR-Cas9 to test a putative fetal globin repressor region, finding it does not autonomously silence gamma-globin, suggesting it is not a therapeutic target for beta-hemoglobinopathies.

Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells achieves curative levels of editing.

This study corrects the sickle mutation in human hematopoietic stem cells using a Cas9 RNP/ssDNA donor approach, showing long-term therapeutic editing in mice.