Zharko Daniloski

LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models

This paper demonstrates the use of LNP-delivered prime editors to correct the PAH R408W mutation in PKU models, achieving ~50% correction efficiency.

Rational design of synthetic proteins using a genome-scale CRISPR screen

The paper designs synthetic proteins for precise gene editing using CRISPR screens and shows improved editing efficiency in various cell types.

Transcriptome-wide profiling of alternative splicing regulators with CRISPore-seq

CRISPore-seq combines CRISPR perturbations with long-read transcriptomics to detect alternative splicing changes, revealing that exon skipping is common and linked to RNA-binding protein perturbations.

Discovery of target genes and pathways of blood trait loci using pooled CRISPR screens and single cell RNA sequencing

The paper identifies target genes and pathways of blood trait loci using CRISPR screens and single-cell RNA sequencing, revealing cis and trans regulatory effects of noncoding variants.

Expanding the scope of bacterial CRISPR activation with PAM-flexible dCas9 variants

This study evaluates PAM-flexible dCas9 variants for bacterial CRISPR activation and repression, finding trade-offs between PAM flexibility and repression efficiency.

Integrative approach identifies SLC6A20 and CXCR6 as putative causal genes for the COVID-19 GWAS signal in the 3p21.31 locus

The paper identifies SLC6A20 and CXCR6 as putative causal genes for the 3p21.31 GWAS signal in COVID-19 using an integrative approach combining CRISPR screens and eQTL data.

An autoimmune disease risk variant has a trans master regulatory effect mediated by IRF1 under immune stimulation

This study identifies a risk variant for autoimmune disease that acts as a trans-eQTL regulating IRF1 under immune stimulation, demonstrating its causal role and mechanism.

The Spike D614G mutation increases SARS-CoV-2 infection of multiple human cell types

The paper shows the D614G mutation in SARS-CoV-2 Spike protein increases infection efficiency in multiple human cell lines by enhancing proteolytic resistance.

Principles for rational Cas13d guide design

The paper develops a computational model to optimize Cas13 guide RNA designs for efficient RNA knock-down, validated across endogenous targets and providing a genome-wide resource for CRISPR screens.