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In a recent article in The Lancet , an international team led by JL Kwiatkowski, F Locatelli and AA Thompson presented their successful multicenter phase 3 clinical trial (HGB-212, NCT03207009) that used gene therapy to treat severe genetic forms of transfusion-dependent -thalassemia

Consistent with this phenotype, our mutants accumulated and secreted high quantities of amino acids and other weak acids (e.g., lactic acid, dicarboxylic acids, keto acids), which strongly alludes to dysregulated cellular metabolism (Fig

The fact that ROS was generated explosively by GSTP-silencing and the incomplete recovery of mitochondrial function by NAC demonstrated in this study indicates that GSTP-silencing induces oxidative stress through mitochondrial dysfunction

Methods The literature search was carried out using research strings in Pubmed, Scopus, and Mendeley via MeSH, with targeted keywords