GaHyun Lee

Frequent, context-dependent effects of human genetic variation on Cas9 activity revealed by population-scale GUIDE-seq-2 and deep combinatorial CHANCE-seq profiling

The paper uses population-scale GUIDE-seq-2 and CHANCE-seq to analyze how genetic variants affect Cas9 activity, finding frequent context-dependent effects on off-target sites.

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.

CHANGE-seq-BE enables simultaneously sensitive and unbiased in vitro profiling of base editor genome-wide activity

The paper introduces CHANGE-seq-BE, a method for sensitive and unbiased profiling of base editor off-target effects in vitro.

Delineating genotypes and phenotypes of individual cells from long-read single cell transcriptomes

The paper introduces scNanoGPS, a toolkit for analyzing long-read single-cell transcriptomes to infer cell genotypes and phenotypes without short-read data, revealing cell-type-specific isoform and mutation patterns in tumors.

The extracellular matrix differentially directs myoblast motility in distinct forms of muscular dystrophy

The paper shows that different forms of muscular dystrophy have distinct ECM compositions that affect myoblast motility, with annexin A6 playing a key role in enhancing movement.