Gene therapy for sickle cell disease (SCD)

Source– This post on Gene therapy for sickle cell disease (SCD) has been created based on the article “India getting close to developing gene therapy for sickle cell disease, say officials” published in “The Hindu” on 20 June 2024.

Why in the news?

India is advancing towards developing a gene therapy for sickle cell disease (SCD).

About CRISPR-Cas9 system

i) India is developing gene therapy using CRISPR-Cas9. It is a gene-editing tool to treat SCD.

ii) The CRISPR-Cas9 system works like molecular scissors. It uses an enzyme to cut DNA at a specific spot, allowing a guide RNA to insert a new genetic code at that location.

iii) This method is considered fast and highly versatile compared to other gene-editing techniques.

iv) Developing a gene therapy using CRISPR has been part of India’s mission to eradicate sickle cell disease by 2047.

About National Sickle Cell Anaemia Elimination Mission

i) The National Sickle Cell Anaemia Elimination Mission is an initiative to address the health challenges posed by sickle cell disease, particularly among tribal populations.

ii) The mission aims to improve care for all Sickle Cell Disease patients and lower the prevalence of the disease through coordinated screening and awareness strategies.

About Sickle cell disease

i) Sickle cell disease is a group of inherited red blood cell disorders affecting haemoglobin.

ii) Normal Cells are of Disc-shaped and flexible, allowing easy movement through blood vessels. In Sickle Cell Disease, Red blood cells become crescent or “sickle” shaped, causing them to be rigid and block blood flow.

iii) Blocked blood flow can lead to serious issues such as Stroke, Eye problems, Infections, Pain crises.

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