GENE EDITING (Syllabus: GS Paper 3 – Science and Technology)

Context: The US Food and Drug Administration (FDA) approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene editing technology.

• The Crispr gene therapy uses the breakthrough gene editing technology that won its inventors the Nobel Prize in 2020.

Genome editing (also called gene editing)

• About: A group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome.
• Approach: A well-known approach to genome editing is called CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9.

oThe CRISPR-Cas9 system is faster, cheaper, more accurate, and more efficient than other genome editing methods.

Sickle cell disease

• About: It is one of a group of inherited disorders known as sickle cell disease.
• Cause: It is caused by a change in the gene that tells the body to make the iron-rich compound in red blood cells called haemoglobin.
• Symptoms: Extreme tiredness or irritability due to anaemia, painful swelling of hands and feet, and jaundice in the early stages.
• Treatment: Blood transfusions alleviate anaemia and lower pain crisis risk.