RNA editing is promising to go where DNA editing can’t
(Source – The Hindu, International Edition – Page No. – 7)
| Topic: GS3 – Science and Technology |
| Context |
| ● A Massachusetts-based biotech company, Wave Life Sciences, has pioneered clinical-level RNA editing to treat genetic disorders, offering a temporary, reversible alternative to DNA editing.
● Unlike DNA edits, RNA modifications do not make permanent changes to the genome, presenting lower immune risks. ● RNA editing holds promise for treating several genetic diseases, though challenges remain. |
Breakthrough in RNA Editing by Wave Life Sciences
- Wave Life Sciences, a biotech firm in Massachusetts, became the first to use RNA editing at the clinical level to treat genetic conditions.
- The success builds on RNA’s essential role in functions like RNA interference and the rapid development of mRNA vaccines, highlighting RNA’s importance beyond gene regulation.
RNA Editing: Mechanism and Technique
- RNA Editing Process: RNA editing corrects mistakes in mRNA after synthesis but before protein formation, preventing faulty proteins associated with genetic disorders.
- ADAR Enzyme: ADAR (adenosine deaminase acting on RNA) converts adenosine to inosine in mRNA, mimicking guanosine’s function and allowing for accurate protein synthesis.
- Guided RNA (gRNA): A gRNA directs ADAR to specific mRNA segments, allowing site-specific RNA editing for treating genetic disorders.
Expanding RNA Editing Applications
- The company aims to treat Huntington’s disease, Duchenne muscular dystrophy, and some forms of obesity using similar RNA editing techniques.
- Other firms, like Korro Bio and Shape Therapeutics, are also working on RNA editing for conditions like Parkinson’s, heart disease, and neurological disorders.
Advantages and Challenges of RNA vs. DNA Editing
- Advantages of RNA Editing:
- Temporary Changes: RNA edits are temporary, allowing effects to fade over time, which helps reduce long-term risks.
- Lower Immune Reaction Risk: Uses ADAR enzymes naturally present in humans, minimising immune responses.
- Reversible Therapy: RNA editing enables therapy discontinuation if issues arise, enhancing patient safety.
- Challenges of RNA Editing:
- Specificity Issues: ADAR enzymes can affect non-targeted areas, risking side effects.
- Repeated Treatments Needed: Effects are temporary, requiring ongoing administration.
- Delivery Constraints: Lipid nanoparticles and vectors limit transport capacity for large molecules.
| Practice Question: Discuss the potential of RNA editing as a therapeutic tool for genetic disorders. Compare its advantages and challenges with DNA editing. (250 Words /15 marks) |
