Gene Therapy Restores Vision in Patients with Rare Genetic Disorder

Source: The Lancet | September 05, 2024

The Breakthrough

Researchers have developed a gene therapy that has successfully restored vision in patients suffering from Leber Congenital Amaurosis type I (LCA1), a rare inherited disorder that causes early-onset blindness.
The findings, published in The Lancet, mark a major advance in the application of DNA technologies for treating genetic diseases.

How the Therapy Works

The treatment uses a virus-based delivery system to introduce a functional gene into the light-sensitive cells of the retina.
Once delivered, the corrected gene enables these retinal cells to respond to light, thereby restoring visual function.

This method demonstrates the practical use of recombinant DNA technology and gene transfer, key themes in Anthropology Paper-I.

Key Findings from the Study

• Light sensitivity in patients improved by up to 10,000-fold after treatment.
• Patients reported significant improvement in reading ability and independent navigation.
• The therapy is designed as a single-dose treatment per eye, with effects expected to be long-lasting or permanent.

Why This Matters

This research highlights the expanding role of gene therapy in human welfare, particularly in treating inherited disorders previously considered incurable.
For Anthropology, it illustrates how advances in human genetics and DNA technologies are directly transforming medical practice.

Future Prospects

Although early results are highly promising, researchers emphasize the need for larger clinical trials to confirm the therapy’s long-term safety and effectiveness.
If successful, this approach could pave the way for treating other monogenic disorders affecting vision and beyond.

Relevant UPSC Anthropology PYQs

1. Describe the practical applications of DNA technology in the current scenario. 15 Marks – 2023
2. What are the applications of human genomic research in human welfare? 15 Marks – 2020
3. Applied human genetics has come to touch every sphere of human life.’ Discuss in light of recent advances in molecular anthropology. 15 Marks – 2019