The First CRISPR Drug Wins U.K. Approval for Sickle Cell Disease

GEN Cutting Edge News

The news recently reported from the GEN (Genetic Engineering & Biotechnology News) said that the U.K. authorities have granted approval to Casgevy, the CRISPR-Cas9 therapy formerly known as exa-cel developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It is a  a landmark announcement for the CRISPR field and the sickle cell disease (SCD) community.he U.S. Food and Drug Administration (FDA) is expected to follow suit in the next three weeks, having issued a decision date of December 8. A further decision on Bluebird bio’s related SCD therapy should come later that month. The U.K. approval was announced by the Medicines and Healthcare products Regulatory agency. The CRISPR-Cas9 technology will offer a cure or more effective treatments for sickle cell disease.

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Picture from website of Genetic Engineering & Biotechnology News

Knowledge About Sickle Cell Disease (SCD)

Sickle cell disease (SCD), also known as sickle cell anemia, is a genetic disorder that affects red blood cells. It is an inherited condition caused by a mutation in the HBB gene, which encodes a component of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. This mutation leads to the production of abnormal hemoglobin known as hemoglobin S (HbS).

In individuals with sickle cell disease, the abnormal hemoglobin causes red blood cells to become rigid, sticky, and crescent-shaped (resembling a sickle). These abnormal cells can get stuck in small blood vessels, leading to various complications, including pain, anemia, organ damage, and a higher risk of infections.

Easy Way For Diagnosing Sickle Cell Disease

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Liuyi Bilotechnology manufactures one model of electrophoresis tank called cellulose acetate membrane elctrophoresis tank DYCP-38C which can be adapted for the analysis of hemoglobin variants, including those associated with sickle cell disease (SCD). It is a traditional and economic method for hemoglobin electrophoresis.  

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Experiment for separating serum protein by Cellulose Acetate Membrane Electrophoresis  

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References

  1. From Genetic Engineering & Biotechnology News: Credit: Artur Plawgo/ Getty Images 

Post time: Nov-21-2023