- Recently, a biotechnology company Wave Life Sciences in the USA became the first company to treat a genetic condition by editing RNA at the clinical level.
- RNA editing fixes errors in messenger RNA (mRNA) during transcription, preventing the production of faulty proteins responsible for genetic disorders.
| Alpha-1 Antitrypsin Deficiency (AATD) It is a genetic disorder caused by mutations in the SERPINA1 gene. It leads to reduced or dysfunctional production of alpha-1 antitrypsin (AAT), a protein that protects tissues from enzyme damage. It primarily affects the lungs, causing emphysema or chronic obstructive pulmonary disease (COPD), and can also damage the liver. Treatments include lifestyle changes, protein replacement therapy, and, in severe cases, liver transplantation. |
- It utilizes adenosine deaminase enzymes (ADAR) paired with guide RNA (gRNA) to correct mutations in mRNA.
- Wave Life Sciences treated alpha-1 antitrypsin deficiency (AATD) using RNA editing to restore normal protein levels.
- It is also developing RNA editing for Huntington’s disease, Duchenne muscular dystrophy, and obesity.
- RNA editing is advantageous over DNA editing as temporary changes reduce long-term risks compared to permanent genome alterations in DNA editing.
- Use of naturally occurring ADAR enzymes minimizes immune reactions.
- Challenges in RNA Editing:
- Non-targeted editing by ADAR can lead to side effects.
- Repeated treatments are needed to sustain therapeutic effects.
- Lipid nanoparticles and viral vectors have restricted carrying capacities.
Dig Deeper: Read about RNA interference.
