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Retinal diseases: RNA therapeutics show promise but is India ready?

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(Source – The Hindu, International Edition – Page No. – 7)

Topic: GS2 – Social Justice – Health, GS3 – Science and Technology
Context
  • The article discusses vision impairment, focusing on inherited retinal diseases (IRDs).
  • It also highlights RNA-based therapies as a promising treatment, emphasizing India’s need for genetic research and precision medicine.

Gene Therapy Breakthroughs

  • In 2017, the U.S. FDA approved the first gene therapy for blindness caused by RPE65 gene mutations.
  • Over 50 clinical trials are exploring gene therapies for IRDs, but awareness in India remains low.
Inherited Retinal Diseases (IRDs) and Their Impact
  • IRDs are genetic disorders that cause gradual vision loss, often leading to blindness.
  • These diseases result from mutations in over 300 genes responsible for retinal function.
  • Some people lose sight at birth, while others experience slow deterioration over time.
  • Early intervention can slow or prevent blindness.
  • About 5.5 million people worldwide have IRDs, with a prevalence of 1 in 3,450 globally.
  • India has a higher prevalence:
    • 1 in 372 in rural South India
    • 1 in 930 in urban South India
    • 1 in 750 in rural Central India

RNA-Based Therapies: A Safer Alternative

  • RNA-based therapies, like antisense oligonucleotides (ASOs), offer temporary, precise treatments without altering DNA.
  • ASOs have successfully treated diseases like spinal muscular atrophy and are now being tested for retinal conditions.
  • Advanced RNA-editing techniques, such as ADAR enzymes and suppressor tRNAs, can correct genetic mutations and restore retinal function.

Precision Medicine in India

    • Precision medicine tailors treatments to genetic makeup, lifestyle, and other factors.
  • India lacks large-scale studies to map IRD mutations, essential for developing targeted therapies.
  • Genetic mutations vary across India’s diverse population, requiring extensive research.

Barriers and Solutions

  • Challenges include low awareness, limited genetic counselling, insufficient funding, and poor diagnostic access in rural areas.
  • Collaboration between research institutions, like CSIR-IGIB and L.V. Prasad Eye Institute, has led to precision therapy development.
  • Partnerships between global and local pharmaceutical companies can improve treatment accessibility.

Conclusion

  • RNA-based therapies offer hope for treating IRDs in India.
  • Prioritizing genetic research, raising awareness, and fostering collaborations are crucial to making these treatments accessible and effective for patients. 
Practice Question:  Examine the potential of RNA-based therapies in treating diseases. Discuss the challenges and strategies for advancing precision medicine in India. (250 Words /15 marks)

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