Genomic variants in the <i>ASS1</i> gene, involved in the Nitric Oxide Biosynthesis and Signaling Pathway, Predict Hydroxyurea Treatment Efficacy in Compound Sickle Cell Disease/β-Thalassemia Patients

Abstract

These markers may exert their effect by inducing nitric oxide biosynthesis, either via altering splicing and/or miRNA binding, as predicted by in silico analysis, and ultimately, increase γ-globin levels, via guanylyl cyclase targeting.