14 August 2024 : Daily Answer Writing
Q1) Describe briefly what gene therapy is and what advantages it has over other treatments?
 (150 Words/10 Marks)
ANS
Gene therapy is a medical technique that uses modified genes to treat, prevent and cure a disease. It involves altering the genes in a patient’s cells to correct the underlying genetic problem.
Advantages of gene therapy over other treatments are as follows:
- Curability: Gene therapy has the potential to cure diseases by repairing faulty genes which is the root cause of a disease. Whereas other treatments like chemotherapy treatments focus also on symptoms or effects of a disease.
E.g., CART-cell therapy cures cancer.
- Permanency: Gene therapy can have long-lasting effects, as the modified genes continue to produce beneficial proteins. This is in contrast with other treatments, such as drugs, which need to be taken repeatedly.
E.g., insulin injections.
- Targeted delivery: Gene therapy interventions can be targeted to specific cells, which can improve the efficiency of treatment. On the other hand, treatments like medication have harmful side effects.
E.g., Radiation therapy has harmful side effects.
- Minimally invasive: Gene therapy products can be delivered through an injection. When compared with surgery, it can reduce the risk of complications.
E.g., Surgery for brain tumour is highly complex.
- Genetic Disorder Treatment: Gene therapy is the most promising treatment for genetic disorders. They are not treatable by standard methods.
E.g., cystic fibrosis, muscular dystrophy, etc.
- Overall cost: Although the treatment might be costly, the reduced chances of relapse results in patient leading a better quality of life, free from frequent medical interventions.
E.g., Cancer recurrence after chemotherapy.
However, gene therapy has certain challenges as mentioned below:
- Unintended changes: There is a risk of immune system rejecting the modified genes can result in long-term side effects.
E.g., University of Cambridge in a study found CRISPR-Cas9 genome editing can lead to unintended mutations in approximately 16% of samples.
- Ethical challenges: Gene therapy technologies can raise ethical considerations as humans intervening with natural affairs (playing God).
E.g., Chinese biophysicist He Jiankui’s designer babies.
- Limited Applicability: Gene therapy may not be effective for complex, multifactorial diseases where the underlying genetic cause is unknown.
E.g., Alzheimer’s disease.
- Accessibility: Its high cost and limited availability can exacerbate the health care divide between rich and poor.
E.g., Treatment of rare diseases can go upto crores of rupees.
Although gene therapy is a revolutionary breakthrough with immense potential it needs to be constantly evolved under a proper regulatory framework. In this regard, the ICMR guidelines for gene therapy in India, 2019 is a step in right direction regarding translational research of gene therapy.
