What is it about?
The article is about the most recognized components in clinical research that are useful for inducing the fetal globin gene expression as an substitute therapy for blood infusion in hemoglobinopathies such as thalassemia (major and intermedia) and sickle cell anemia. It argues that by using two or more components together in one experiment the results would be more effective than using just one inducer. The previous clinical results shows that the induced fetal globin gene in adult patients might decline over the course of therapy.
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Why is it important?
Considering the synergistic effect of two or more component in therapy of people with thalassemia and sickle cell anemia, will cover the problem of losing the effect of treatment over the course of therapy. The previous findings from clinical outcomes show that the induction of fetal globin gene as a new substitution therapy for blood infusion in people with thalassemia and SCD is not satisfying to induce the gene for short time and it must be effective for long time and on the other hand the combination of treatments must be safe.
Perspectives
It calls scientists from bioinformatics and molecular biology to design experiments in silico to examine the multiple effects of Hb F inducers prior to clinical tests to find the best match of inducers which are potent and safe during the long time of therapy. The future combination of these components could safely replace blood infusion in patients with hemoglobinopathies.
PEGAH ADELVAND
Pasteur Institute of Iran
Read the Original
This page is a summary of: The intrinsic genetic and epigenetic regulator factors as therapeutic targets, and the effect on fetal globin gene expression, Expert Review of Hematology, December 2017, Taylor & Francis,
DOI: 10.1080/17474086.2018.1406795.
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