RNA-based therapies use RNA molecules to modulate biological pathways, offering temporary, precise treatments without altering DNA. Applications include correcting genetic mutations (ADAR enzymes), bypassing stop-codon mutations (suppressor tRNAs), and treating diseases like cystic fibrosis (PTC124) and spinal muscular atrophy (ASOs). India needs genetic research and infrastructure to adopt these therapies for inherited retinal diseases.
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According to the World Health Organisation, more than 2.2 billion people worldwide experience some form of vision impairment, including inherited retinal diseases (IRDs).
Inherited retinal diseases (IRDs) are a group of genetic conditions that affect the retina and can lead to vision loss or blindness. IRDs are rare but can affect people of all ages.
Symptoms Difficulty seeing in the dark or dim, Reduced side vision, Glare and difficulty in bright light, Reduced central vision, and Difficulty seeing colors or differences between colors.
RNA-based therapies offer a precise, temporary way to correct genetic defects without altering DNA.
Approaches like antisense oligonucleotides (ASOs), ADAR RNA editing, suppressor tRNAs, and PTC124 bypass mutations or restore protein production in retinal cells.
These methods avoid risks associated with permanent DNA changes and have already shown success in treating spinal muscular atrophy and cystic fibrosis.
Global advancements are encouraging, however the country lacks large-scale genetic studies to map IRD mutations in its diverse population.
Without data on prevalent mutations—like the ABCA4 gene’s role in Indian subgroups—researchers cannot design targeted therapies.
Limited awareness among clinicians, scarce genetic counseling, and uneven diagnostic infrastructure further hinder progress.
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PRACTICE QUESTION Q.In the question given below, there are two statements marked as Assertion (A) and Reason (R). Assertion: RNA-based therapies are considered safer than DNA-based therapies. Reason: RNA-based therapies make temporary changes that do not alter the genome permanently. Which of the options given below is correct? A) Both A and R are true, and R is the correct explanation for A. B) Both A and R are true, but R is not the correct explanation for A. C) A is true, but R is false. D) A is false, but R is true. Answer: A Explanation: The assertion is true: RNA-based therapies are safer than DNA-based therapies. The reason is also true and correctly explains the assertion: RNA-based therapies make temporary changes and do not alter the genome permanently. |
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