Article
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria.
Leukemia - 1 Jul 2026
Mizumaki Hiroki, Gao Shouguo, Wu Zhijie, Gutierrez-Rodrigues Fernanda, Alemu Lemlem, Quinones Raffo Diego, Darden Ivana, Rios Olga, Lotter Jennifer, Kajigaya Sachiko, Durrani Jibran, Groarke Emma M, Patel Bhavisha A, Young Neal S
Abstract excerpt
Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-mutated cells occurs uniquely in the setting of bone marrow (BM) failure, but specific pathophysiologic mechanisms remain unclear. We performed single-cell RNA sequencing (scRNA-seq) of BM cells from patients with...
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