Hyderabad: While stem cell therapy is hailed as one of the most exciting treatment methods of the modern age, gene therapy is also equally promising, though it has many detractors. Scientists have shown how gene therapy can be used to edit or replace genome on a small scale. But the concept is still grappling with issues related to efficiency and safety.
Recently, a Harvard University research team claimed that it had edited the genome of mice in a bid to safeguard them against heart disease.
Researchers claimed they had cut down the risk of heart disease by almost 90 per cent. The research team, led by Dr Kiran Musunuru from the Harvard University Department of Stem Cell and Regenerative Biology and Brigham and Women’s Hospital published the results in Circulation Research. The mice used had a predisposition to heart disease due to a genetic mutation. Scientists engineered a virus with a DNA-disruptive mechanism and injected it into the liver of the mice.
“We found that within three to four days of administration of the virus, the mutagenesis rate of Pcsk9 (the mutation) in the liver was as high as 50 per cent,” Dr Musunuru and his team reported. Cholesterol levels were found to be 50 per cent lesser in the blood of the mice.
Several studies in AP and South Indian states have shown mutations that exist in our populations make us vulnerable to heart diseases and diabetes.
Some of these are found in the North Indian states and European countries, while some are not. Genome editing techniques could come in handy as a one-time cure without the need for surgeries.
However, the therapy will still take a lot of time to be approved. Dr Col M. Sitaram, president, AP Cardiological Society of India, said, “Gene therapy has a long way to go. We are still not equipped to carry out genome editing.
However, gene mapping is something that is picking up. We can look for mutations in the genome of a person and tell which disease he is vulnerable to.”
