India is getting closer to developing a gene therapy for sickle cell disease.
- Developing a gene therapy using CRISPR has been part of India’s mission to eradicate sickle cell disease by 2047.
- World Sickle Cell Day is celebrated on June 19 to raise awareness about this genetic blood disorder and to educate people about how to manage their lifestyles.
About Sickle Cell Disease
Ite is a genetic blood disorder with a high prevalence rate among the Scheduled Tribes.
- It is a group of inherited red blood cell disorders.
- Red blood cells contain haemoglobin, a protein that carries oxygen.
- Healthy red blood cells are round. SCD affects the shape of red blood cells, which carry oxygen to all parts of the body.
- It causes the red blood cells to become hard and sticky and look like a C-shaped farm tool called a “sickle.”
- Sickle cells die early, which causes a constant shortage of red blood cells.
- Also, when they travel through small blood vessels, they get stuck and clog the blood flow.
- Threats Posed: Increases the risk of infection and of death from conditions like stroke, heart problems, kidney problems, and pregnancy complications.
- Cure:
- Only cure comes in the form of gene therapy and stem cell transplants — both costly and still in developmental stages.
- Blood transfusion, wherein red blood cells are removed from donated blood and given to a patient, is also a trusted treatment in the absence of permanent cures
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About CRISPR-Cas9
- CRISPR stands for ‘Clustered Regularly Interspaced Short Palindromic Repeats’.
- Cas9 stands for CRISPR-associated protein 9, & is the nuclease part that cuts the DNA.
- CRISPR is the DNA-targeting part of the system which consists of an RNA molecule, or ‘guide’, designed to bind to specific DNA bases through complementary base-pairing.
- The CRISPR-Cas9 system was originally discovered in bacteria that use this system to destroy invasions.
- It is the most common, cheap & efficient system used for genome editing.
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Casgevy And Lyfgenia: CRISPR-Based Gene Therapies
- Casgevy made by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia, by Bluebird Bio—is for people 12 and older.
- These two therapies work in different ways however both therapies utilise the Nobel-winning CRISPR/Cas 9 genome editing technology.
What Is Casgevy Therapy?
- The Casgevy therapy uses the patient’s blood stem cells, which are precisely edited using Crispr-Cas9.
- The therapy targets a gene called BCL11A, which is crucial for switching from fetal to adult haemoglobin.
- The therapy uses the body’s mechanisms to produce more foetal haemoglobin, alleviating the symptoms of the two conditions
What is Lyfgenia Therapy?
- It uses a viral envelope to deliver a healthy hemoglobin-producing gene.
- Lyfgenia works by taking a piece of a virus (a lentivirus, which belongs to the HIV family) and using it to deliver a functional version of a haemoglobin-producing gene.
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(Know more about Casgevy And Lyfgenia: CRISPR-Based Gene Therapies, here)
Government Initiatives to address SCA
Why Prevalent in Tribal Population?
- Tribal areas were endemic to malaria for many years, leading to many deaths, thus, as an evolutionary, their RBCs were becoming sickle shaped.
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- National Sickle Cell Anaemia Elimination Mission: Introduced in the Union Budget 2023, focuses on addressing the significant health challenges posed by sickle cell disease, particularly among tribal populations of the country.
- Implemented in 17 high-focus states across the country, this program aims to improve the care and prospects of all sickle cell disease patients while reducing the prevalence of the disease.
- The program is executed in a mission mode as part of the National Health Mission (NHM), aims to eliminate sickle cell genetic transmission by the year 2047, showing a long-term commitment to eradicating the disease.
- Budget 2023-24: Government announced its plans to distribute “special cards” across tribal areas to people below the age of 40.
- The cards will be divided into different categories based on the screening results.
- The mission will receive funding under the National Health Mission.
- The Ministry of Tribal Affairs (MoTA) has launched the Sickle Cell Disease Support Corner: To bridge the gap between patients and health care services in tribal areas.
- The Portal provides a web-based patient powered registration system which collates all information related to Sickle Cell Disease (SCD) among tribal people in India, including providing them a platform to register themselves if they have the disease or the trait.
- National Council on Sickle Cell Disease has also been constituted of senior officials of GoI and health care private and public bodies for timely and effective action.
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June 21, 2024
