How can gene editing help cure diseases? | In Focus podcast

This month, there’s been a lot of excitement in the medical world over the approval given to treatments for diseases that based on genome editing. What’s this all about? In 2020, the Nobel Prize for Chemistry was given to two women scientists, Emmanuelle Charpentier and Jennifer Doudna for their discovery of what is essentially a genetic scissors, a tool that allows scientists to cut specific sites of a human being’s DNA, or to edit it, by making minor changes.

This tool, known as the CRISPR/Cas9 system, opened up opportunities to treat certain genetic or inherited disorders. Two of these are blood disorders, beta thalassemia and sickle cell disease, up until now could only be cured through bone marrow transplants. Now, they can potentially be cured by editing the patient’s own genes.

In the Union Budget this year, Finance Minister Nirmala Sitharaman had announced a sickle cell anaemia mission - to eliminate the condition by 2047. India is the second-worst affected country in terms of predicted births with sickle cell anaemia.

As exciting as these new developments sound, they will likely be extremely expensive and therefore, probably unaffordable to many. Also, the clinical trials, have at present, only evaluated a small number of patients for relatively short durations and there is a need to constantly monitor the safety and efficacy of these therapies.

So what exactly does genome editing involve? Can its potential be expanded to treat far more diseases, and what lies ahead in this field? What are the concerns surrouding this - could there be unintended consequences to genetic modifications?