CRISPR for Sickle Cell Anaemia
Source: Indian Express
GS III: Science and Technology
What is discussed under CRISPR for Sickle Cell Anaemia?
- What are genome editing and CRISPR-Cas9?
- Sickle Cell Anaemia (SCD)
Why in News?
India has given the go-ahead for a five-year effort to create CRISPR technology to treat sickle cell anaemia.
What are genome editing and CRISPR-Cas9?
Genome editing
- Genome editing refers to a range of scientific techniques that enable the modification of an organism’s DNA.
- At specific sites in the genome, these technologies enable the addition, removal, or modification of genetic material.
- The prevention and treatment of human diseases is a major area of focus for genome editing.
Applications of genome editing technologies
- Better understanding of diseases
- Identifying threats
- Developing new treatments
- Treating genetic diseases
CRISPR-Cas9
- CRISPR-Cas9 is a genome editing approach which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9.
Image by Crystal Blair from Pixabay - A naturally occurring genome editing system that bacteria deploy as an immunological response is a basis for CRISPR-Cas9.
- Bacteria that are virus-infected seize tiny bits of the viruses’ DNA and splice them into their own DNA in a specific pattern to form sections known as CRISPR arrays.
- The bacteria can “remember” the viruses thanks to the CRISPR arrays (or closely related ones).
- In the event of a subsequent virus attack, the bacteria create RNA segments from CRISPR arrays that can recognise and bind to particular sections of the viral DNA.
- The virus is then rendered inoperable by the bacteria’s employment of Cas9 or a related enzyme to split the DNA.
- Researchers adapted this immune defence system to edit DNA.
Ethical Dilemma
- The capability of CRISPR to cause profound alterations in a person can be abused.
- Designer Baby: In 2018, a Chinese scientist revealed that he had changed a human embryo’s genes to prevent HIV infection.
- Since this was the first known instance of a “designer baby,” the scientific community was deeply troubled.
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- A population of superhumans could be created if changes in the embryo are passed down through the generations.
- When therapeutic treatments are used, the altered genetic sequences are retained by the individual and are not inherited by the offspring.
Sickle Cell Anaemia (SCD)
- A collection of genetic red blood cell diseases is known as sickle cell disease (SCD).
- Haemoglobin is an oxygen-carrying protein found in red blood cells. Round and healthy red blood cells travel via tiny blood channels to deliver oxygen to every region of the body.
- In a person with SCD, the haemoglobin is defective, which makes the red blood cells stiff and sticky and resemble the “sickle,” a C-shaped farm implement.
- Red blood cells are constantly in insufficient supply due to the early death of sickle cells.
- Additionally, they become caught and obstruct the flow of blood when they pass through tiny blood arteries.
- Infection, acute chest syndrome, stroke, and other severe consequences (health issues) might result from this, in addition to pain.
- SCD patients may begin to exhibit symptoms in the first year of life, typically around the age of five months.
- Each person will experience SCD differently, with varying degrees of moderate to severe symptoms and problems.
- A bone marrow or stem cell transplant is the only treatment recognised by the FDA as having the potential to treat SCD.
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