RNA Therapeutics for Retinal Diseases: India’s Preparedness

Why in the news?

RNA-based therapies show promise for treating Inherited Retinal Diseases (IRDs), but India faces challenges due to limited research, awareness, and genetic mapping. Collaborative efforts and genetic studies are essential to make these therapies accessible to Indian patients.

RNA Therapeutics for Retinal Diseases: India’s Preparedness

Current Landscape of Inherited Retinal Diseases (IRDs):

  • Prevalence: IRDs cause progressive vision loss, leading to blindness. Globally, 5.5 million people are affected, with a higher prevalence in India (1 in 372 in rural South India).
  • Genetic Causes: Over 300 genes are linked to IRDs, causing mutations in the retina. Early intervention can slow or prevent blindness.
  • Need for Research: There is a lack of large cohort studies in India (e.g., 500+ patients) to understand mutation spectra of IRDs, essential for precision medicine development.

RNA-Based Therapeutics: A Promising Approach

  • Gene Therapy: While gene therapy like RPE65 has shown promise, it’s not universal for all genetic eye diseases.
  • RNA Therapeutics: RNA-based therapies such as antisense oligonucleotides (ASOs) and RNA-editing with ADAR enzymes are being explored for treating IRDs like Stargardt disease and Leber congenital amaurosis.
  • PTC124: A small molecule RNA therapy, currently used for cystic fibrosis, is being trialed for aniridia, another eye disorder.
  • These therapies offer targeted, personalized treatments, improving outcomes for IRD patients.

India’s Readiness for RNA-Based Precision Therapies

  • Genetic Research Gap: India lacks large-scale genetic research to understand prevalent IRD mutations and design precision therapies.
  • Challenges: Barriers include limited awareness, genetic counseling, diagnostic infrastructure, and research funding, particularly in rural areas.
  • Collaborations: A June 2024 study between CSIR-Institute of Genomics and L.V. Prasad Eye Institute has advanced precision therapies for IRDs.
  • Way Forward: More research, global-local collaborations, and clinician awareness are essential for advancing RNA-based therapies and making them accessible to Indian patients.

About RNA Therapeutics :

  • Definition: RNA therapeutics are medicines that use RNA molecules to regulate, repair, replace, or enhance gene expression.
  • Mechanisms:
  • mRNA: Provides instructions for cells to produce specific proteins (e.g., vaccines).
  • siRNA: Targets and silences specific genes, reducing undesirable protein production.
  • ASOs: Bind to messenger RNA to modify gene expression, often used in genetic disorders.
  • Promise: RNA therapies hold potential for treating genetic diseases by targeting the root cause at the molecular level.

Sources Referred:

PIB, The Hindu, Indian Express, Hindustan Times