GenomicsbioRxivPreprint
Single-cell genotype-phenotype mapping identifies therapeutic vulnerabilities in VEXAS syndrome
Saravanan Ganesan, Rebecca M. Murray, Jesús Alfonso López Sotélo…·Dan Avi Landau
This study uses single-cell multi-omics to map genotype-to-phenotype relationships in VEXAS syndrome, identifying PERK pathway activation as a therapeutic target.