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Sickle Cell Disease: CRISPR Gene Therapy on the Horizon



  Nov 06, 2023

Breakthrough in Sickle Cell Disease Treatment



Sickle Cell Disease (SCD) Overview:

Sickle Cell Disease is a genetic disorder causing abnormally shaped red blood cells that can lead to severe health complications. Affected individuals suffer from symptoms like pain, infections, stroke, and other serious issues due to the crescent-shaped cells disrupting blood flow.

Potential Gene Therapy Cure:

Upcoming FDA Review: A gene therapy, possibly the first to use CRISPR in the US, is under review by FDA advisors.

Treatment Mechanism: The therapy aims to edit the DNA in blood cells to produce healthy haemoglobin, potentially addressing the root cause of SCD.

Current Status: Early results are promising, showing pain relief and reduced hospital visits for patients.

Safety and Approval Process:

Expert Consultation: The FDA will consult with gene therapy experts to evaluate potential risks and long-term safety.

Post-Approval Research: If approved, the company plans to conduct safety monitoring and further research.

Economic Considerations:

Cost Analysis: Costs are undisclosed but could be offset by reducing the financial burden of current SCD treatments.

Indian Health Mission: India’s goal to eliminate sickle cell anaemia by 2027 highlights the global importance of affordable and accessible cures.

This potential gene therapy could mark a significant milestone in the treatment of SCD, providing a one-time, long-term solution for those affected by this debilitating condition.

SRIRAM’s


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