RNA Editing vs DNA Editing:

• DNA editing (CRISPR/Cas9): Makes permanent changes to a person’s genome, leading to irreversible outcomes. If errors occur, it can have long-term effects.
• RNA editing: Temporary changes that can be reversed or stopped. This makes it safer for clinical use, as the therapy can be stopped if issues arise.

Key Concepts of RNA Editing:

• RNA interference: Small RNA molecules prevent gene expression, playing a vital role in medical advances, such as the success of mRNA vaccines during COVID-19.
• Messenger RNA (mRNA): Synthesized using DNA instructions, mRNA helps in translating genetic information into proteins. It is a key area where RNA editing can alter the instructions without changing the DNA.

What is RNA Editing?

• Cell processes: Cells transcribe DNA into mRNA, which carries instructions to produce proteins.
• Adenosine deaminase acting on RNA (ADAR): A group of enzymes that convert adenosine into inosine in RNA, altering the instructions temporarily.
• Application: Can correct mutations or defects at the RNA level, beneficial for diseases where the problem lies in protein production.

Advantages of RNA Editing:

• Specificity: Can precisely target specific parts of mRNA to correct or alter protein production without permanent genome modification.
• Reversibility: Allows for easier correction of potential errors, offering a safer therapeutic approach.

RNA Editing in Development:

• Wave Life Sciences: Working on RNA editing for diseases like Duchenne Muscular Dystrophy and obesity. They are also looking to treat conditions like AATD (Alpha-1 Antitrypsin Deficiency), which leads to lung and liver diseases.
• ADAR enzymes: Being used to edit mRNA and correct harmful mutations. Companies like Eli Lilly, Roche, and Novo Nordisk are involved in developing RNA editing technologies.

Challenges in RNA Editing:

1. Efficiency: Current methods must achieve high efficiency to correct defects effectively.
2. Immune Response: Delivery mechanisms like lipid nanoparticles are used, but potential immune system reactions are still a concern.
3. Durability: Maintaining the edited RNA’s function over time without requiring continuous intervention is crucial.