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Gene Therapy to cure Cancer

  • Published
    15th Dec, 2022

Scientists in the United Kingdom testing a new form of cancer therapy, reported success in a teenage girl, Alyssia, with a form of cancer called T-cell acute lymphoblastic leukaemia.

  • Alyssia was the first to receive experimental gene therapy that relied on a new technique called ‘base editing.’

What is T-cell acute lymphoblastic leukaemia?

  • In this form of blood cancer, the T-cells, which are a class of white blood cells, equipped to hunt and neutralise threats to the body, turn against the body and end up destroying healthy cells that normally help with immunity.
  • The disease is rapid and progressive and is usually treated by chemotherapy and radiation therapy.

What is ‘base editing?’

  • A person’s genetic code is several permutations of four bases:
    • Adenine (A)
    • Guanin (G)
    • cytosine (C)
    • thymine (T)
  • Sequences of these bases, akin to letters in the alphabet, spell out genes that are instructions to produce the wide array of proteins necessary for the body’s functions.
  • In the last two decades, the world of biomedical engineering has been enthused by a technique that allows genes to be altered and errors ‘fixed.’
    • The most popular among these approaches has been the CRISPR-cas9 system.
      • Inspired by how certain bacteria defend themselves against viruses, by snipping out and storing pieces of their genes, the CRISPR-Cas 9 system, consists of an enzyme that acts like molecular scissors.
      • It can be made to cut a piece of DNA at a precise location and a guide RNA can be used to insert a changed genetic code at the sites of the incision.
      • While there are a few ways to effect such changes, the CRISPR-cas9 system is believed to be the fast, most versatile system to effect such gene editing.
    • While still a nascent technology, base editing is reportedly more effective at treating blood disorders which are caused by so-called single point mutations, or when a change in a single base pair can cause terminal disease.
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