Congyi Lu

CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder

This study shows that CRISPR activation can rescue electrophysiological deficits in SCN2A haploinsufficiency by increasing the expression of the remaining functional allele in mice and human neurons.

Paired CRISPR screens to map gene regulation in cis and trans

The paper uses paired CRISPR screens to identify cis- and trans-regulatory elements controlling PD-L1 expression in PDAC cells and links them to clinical outcomes.

Enhancer-targeted CRISPR-A rescues haploinsufficiency and mutant phenotypes in organoid models of autism

This study uses CRISPR-A to enhance expression of CHD8 and SCN2A in organoids, rescuing haploinsufficiency and associated phenotypes in ASD models.

Pluripotent stem cell derived models of neurological diseases reveal early transcriptional heterogeneity

The paper uses scRNA-seq to show increased transcriptional heterogeneity in neurodegenerative disease models, suggesting early regulatory imbalances.

The BTB transcription factors ZBTB11 and ZFP131 maintain pluripotency by pausing POL II atpro-differentiation genes

A CRISPR screen identifies ZBTB11 and ZFP131 as factors maintaining pluripotency by pausing POL II at pro-differentiation genes.