CRISPR for Sickle Cell Anaemia

CRISPR for Sickle Cell Anaemia
Source: Indian Express
GS III: Science and Technology

What is discussed under CRISPR for Sickle Cell Anaemia?

India has given the go-ahead for a five-year effort to create CRISPR technology to treat sickle cell anaemia.

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

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.

- 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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