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Increasing Fetal Hemoglobin may be the Key to Help People With Sickle Cell Disease and Thalassemia
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Increasing Fetal Hemoglobin may be the Key to Help People With Sickle Cell Disease and Thalassemia

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Highlights:
  • An important signaling protein which regulates the production of hemoglobin has been identified
  • CRISPR-Cas9 genetic screen was used to look for fetal hemoglobin regulators in human red blood cells, which could become a potential novel drug to treat Sickle Cell Disease (SCD) and Beta-Thalassemia

An important signaling protein which regulates the production of fetal hemoglobin. Increasing the amount of fetal hemoglobin can potentially be therapeutic for Sickle Cell Disease (SCD) and Beta-Thalassemia, reveals a new medical research published by researchers at the Children's Hospital Philadelphia and the Perelman School of Medicine, University of Pennsylvania.

Fetal hemoglobin acts as the main oxygen transport protein during the last seven months of fetus development in the uterus.

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Hemoglobinopathies like Sickle Cell Disease (SCD) and Beta-Thalassemia are characterized by a quantitative reduction in hemoglobin levels or sickling of cells as in the case of SCD. Research efforts are on to find a therapeutic to tackle the lack of hemoglobin production.

There is already sufficient evidence that higher levels of fetal hemoglobin reduce the severity of the disease in SCD. Drugs like hydroxyurea and pomalidomide are currently in use and work by increasing the quantity of fetal hemoglobin. However, these drugs do not work uniformly for all patients and there is a clear need for novel drugs.

Enzyme Acts as a Novel Therapy for Blood Disorders

The research team used CRISPR-Cas9 genetic screen to look for fetal hemoglobin regulators in human red blood cells which could become a potential novel drug to treat Sickle Cell Disease (SCD) and Beta-Thalassemia.
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The genetic screen was able to locate the heme-regulated inhibitor (HRI) known as EIF2AK1 which plays a significant role in repressing fetal hemoglobin. They found that blocking this protein can increase fetal hemoglobin production and this can deliver therapeutic benefits to blood disorders. This discovery clarifies the puzzle about the factor blocking fetal hemoglobin production.

When the research team silenced HRI they were able to increase fetal hemoglobin. Significantly, the team also found silencing HRI decreased the rate of sickling in red blood cells.

Study co-leaders Gerd A. Blobel, MD, PhD and Junwei Shi, PhD also engaged in experiments to indicate proofs-of-concept to see if a potential novel drug to knockout HRI would be more effective in a combination therapy with other drugs like pomalidomide.

The research team found that a combination of HRI knockout and pomalidomide had a positive effect on the overall production of fetal hemoglobin rather than using each therapy separately.

The team believes that this combination therapy could work well for certain mutations of Beta-Thalassemia.

Long-term clinical studies are needed for further evaluation of therapeutic value for this novel line of treatment.


Reference :
  1. Grevet, Jeremy D., Xianjiang Lan, Nicole Hamagami, Christopher R. Edwards, Laavanya Sankaranarayanan, Xinjun Ji, Saurabh K. Bhardwaj et al. "Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells." Science 361, no. 6399 (2018): 285-290.


Source: Medindia

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