Gang Ning

The paper investigates the biological impact of GBA1 mutations using multi-omics in isogenic iPSC lines, finding limited overlapping results across cell types and suggesting RNA-based quantitation may not be optimal for characterizing GCase mechanisms.

This paper improves Cas9 RNP editing in human stem cells to achieve high rates of biallelic homology-directed repair of SNVs by using dCas9 to block re-cutting and damage.