John P. Manis

CRISPRLungo is a computational pipeline for analyzing long-read CRISPR experiments, improving accuracy in detecting structural variants and small indels through UMI-based error correction and benchmarking.

This paper discovers and characterizes SH6, a small molecule degrader of ZBTB7A that increases fetal hemoglobin expression in erythroid cells and shows therapeutic potential for β-hemoglobinopathies.

This study identifies BM adipocyte-derived Resistin, regulated by RXR signaling, as critical for HSC self-renewal and hematopoietic regeneration after myeloablation.

This study shows that gene editing of HSCs without ex vivo culture reduces genotoxicity while maintaining therapeutic efficacy for beta-hemoglobinopathy treatment.