RNA
EDITING: SCIENCE & TECHNOLOGY
NEWS: RNA editing is
promising to go where DNA editing can’t
WHAT’S
IN THE NEWS?
Wave Life Sciences has pioneered clinical RNA editing,
offering a temporary and reversible treatment for genetic disorders by
modifying mRNA without altering DNA. This approach reduces immune risks but
requires ongoing treatment due to its non-permanent effects.
Breakthrough
in RNA Editing by Wave Life Sciences
- Wave Life Sciences, a Massachusetts-based
biotech, has achieved the first clinical-level RNA editing to treat
genetic disorders.
- RNA editing offers a temporary
and reversible alternative to DNA editing, reducing immune risks.
How
RNA Editing Works
- RNA Editing Process: Corrects errors in mRNA,
preventing faulty protein production associated with genetic disorders.
- ADAR Enzyme: ADAR (adenosine deaminase
acting on RNA) converts adenosine to inosine in mRNA, helping to
produce accurate proteins.
- Guided RNA (gRNA): Directs ADAR to specific
mRNA regions, enabling targeted corrections.
Applications
and Future Potential
- Targeted Diseases: The company focuses on
conditions like Huntington's disease, Duchenne muscular
dystrophy, and certain obesity types.
- Other biotech firms are
exploring RNA editing for conditions like Parkinson’s, heart
disease, and neurological disorders.
RNA
Editing vs. DNA Editing: Pros and Cons
Advantages:
- Temporary Changes: Effects are short-term,
allowing flexibility in treatment.
- Reduced Immune Risks: Uses natural human ADAR
enzymes, lowering immune reactions.
- Reversible Therapy: Therapy can be halted if
issues arise, improving safety.
Challenges:
- Specificity: Risk of affecting
unintended areas, potentially causing side effects.
- Repeat Treatments: Temporary effects require
ongoing administration.
- Delivery Limitations: Challenges in delivering
RNA treatments due to size and transport methods.
Source:
https://www.thehindu.com/sci-tech/science/rna-editing-promises-to-go-where-dna-editing-cant/article68836223.ece