Single-cell genotype-phenotype mapping identifies therapeutic vulnerabilities in VEXAS syndrome
Saravanan Ganesan, Rebecca M. Murray, Jesús Alfonso López Sotélo, Elliot O. Eton, Kouhei Takashima, Theo Botella, Kai Beattie, Alyssa C. Indart, Nada Chraiki, Carolyne Croizier, Franco Izzo, Catherine J. Potenski, Samuele G. Marro, Zhijie Wu, Shouguo Gao, , 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.
bioRxiv · May 19, 2024 · PreprintGenomics