TranSYS ESR Latest publication

TranSYS ESR 14 Kariofyllis Karamperis, at the Golden Helix Foundation, announces latest publication on ‘Genome-based therapeutic interventions for β-type hemoglobinopathies’ published in Human Genomics.

Existing treatments in patients with β-type hemoglobinopathies are usually lacking therapeutic efficacy and therefore the research interest has focused on different directions and strategies. Nowadays, the development of current and new technologies has led to the direct and in-direct integration of genome-based therapeutic interventions to both drug and gene therapy, respectively. In the first case, pharmacogenomics, acting as genome-guided treatment, can potentially differentiate the patients to hydroxyurea response and hence ameliorate the disease severity. In terms of gene therapy, the upgrade of intervention tools along with gene addition and genome editing technologies has significantly contributed to the optimization of the whole process, thus demonstrating a series of benefits in clinical trials. The purpose of this article is to shed light on pharmacogenomics, gene addition, and genome editing technologies, and consequently, their potential use as direct and indirect genome-based interventions, in different strategies, referring to drug and gene therapy. Last but not least, all the recent progress, updates, and scientific achievements for patients with β-type hemoglobinopathies are highlighted.

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Author: Kariofyllis Karamperis

ESR14: Standardization of disease and population-specific genotyping panel for preemptive pharmacogenomics: Towards population pharmacogenomics