Why in news
Wave Life Sciences, a biotechnology company, made headlines on October 16 for becoming the first to treat a genetic condition by editing RNA at the clinical level.
The treatment used RNA editing to address a genetic disorder called -1 antitrypsin deficiency (AATD), marking a breakthrough in precision medicine.
What is RNA Editing?
RNA editing involves correcting mistakes in the mRNA sequence after it’s made by the cell but before it’s used to produce proteins.
Adenosine deaminase acting on RNA (ADAR) enzymes convert adenosine in mRNA to inosine, which mimics guanosine and helps correct faulty sequences.
This process helps restore the function of mRNA and ensure normal protein production
RNA Editing in Development
Wave Life Sciences is using RNA editing to treat AATD, which causes protein buildup affecting the liver and lungs.
Their therapy WVE-006 targets single-point mutations in the SERPINA1 gene.
RNA editing is also being explored for Huntington’s disease, Duchenne muscular dystrophy, and forms of obesity.
Other companies like Korro Bio, ProQr Therapeutics, and Shape Therapeutics are developing RNA editing for various conditions like Parkinson’s and heart disease.
RNA vs. DNA Editing
RNA Editing:
Makes temporary changes to mRNA, allowing effects to wear off over time, reducing long-term risks.
Uses ADAR enzymes, which naturally exist in the human body, lowering the risk of immune reactions.
DNA Editing:
Causes permanent changes to the genome, which can lead to irreversible errors.
Requires bacterial proteins, which may cause immune reactions in some cases.
Challenges in RNA Editing
ADAR enzymes may affect non-target regions or miss the intended target, causing side effects.
RNA editing requires repeated treatments as its effects are temporary.
The delivery methods, such as lipid nanoparticles or AAV vectors, have limited capacity for transporting large molecules.
Market Value and Future Outlook
RNA editing is still in its early stages, with 11+ biotech companies exploring its potential for treating various diseases.
Large pharmaceutical companies like Eli Lilly, Roche, and Novo Nordisk have shown interest.
As research advances, RNA editing is expected to become a regular part of clinical gene-editing treatments in the future.
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