Treatment that is tailored just for you

Can personalised medicine and gene editing be the future of healthcare? Experts weigh in on the medical model that helps identify the root cause of an illness at a molecular level

- GARIMA SADHWANI

A NEW STUDY, published in The New England Journal of Medicine in May this year, brought to light a unique case involving a 7-month-old baby, known as 'KJ', who was born with a life-threatening liver disorder known as carbamoyl phosphate synthetase 1 (CPS1) deficiency.

In the case, reported in February this year, it was for the first time that a gene-editing CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-based technology was used with a specific mutation in a living person. While the baby wasn't fully cured, the treatment - a historic step towards personalised medicine - was initiated to ensure that any risks associated with the condition are minimised.

Explaining what personalised medicine is, Dr Shrinidhi Nathany, consultant, molecular haematology and oncology, Fortis Memorial Research Institute, Gurugram, explains, "Personalised medicine refers to a transformative approach in healthcare that uses an individual's genetic, molecular, and clinical profile to guide diagnosis and treatment."

Dr Mandeep Singh Malhotra, director of surgical oncology at the CK Birla Hospital, Delhi, explains, "Instead of using a one-size-fits-all method, personalised medicine involves evaluating the patient's DNA and RNA to detect any aberrations in the genome that may be causing the disease. This helps in identifying the root cause of an illness, whether it's a rare genetic disorder, cancer, or another condition, at a molecular level."

The specific treatment method used in this case was gene-editing. Dr Sajjan Rajpurohit, senior director of medical oncology at BLK-Max Super Speciality Hospital, says, "Gene editing refers to a set of technologies that allow scientists to modify an organism's DNA. One of the most well-known gene-editing techniques is CRISPR-Cas9, which enables precise alterations to specific genes. This technology has opened new avenues for treating genetic disorders" by correcting mutations at their source."