RNA Therapy
30-01-2025
09:04 AM

RNA Therapy Latest News
In recent days RNA-based precision therapeutics are emerging as a game-changer for genetic disorders, including inherited retinal diseases (IRDs).

About RNA Therapy
- It is a term used to describe the use of RNA-based molecules to modulate biological pathways to cure a specific condition.
- Unlike DNA or genome-editing therapies, RNA-based therapies offer a safer alternative as they make temporary changes that don’t carry over to future generations, reducing the risk of unintended long-term effects.
Applications of RNA based Therapies
- RNA-editing with ADAR enzymes: It can correct specific genetic mutations at the RNA level. This method has the potential to restore protein production in retinal cells without altering the underlying DNA, offering a new way to treat retinal degenerative diseases caused by single-point mutations.
- Using of suppressor tRNAs: It is to bypass stop-codon mutations, which can prematurely halt protein synthesis in retinal cells. By enabling the production of full-length proteins, this approach could help restore proper retinal function in IRD patients.
- PTC124 Method: It is also known as ataluren, which is already being used to treat patients with cystic fibrosis and Duchenne muscular dystrophy.
- Antisense oligonucleotides (ASOs) have already been used successfully to treat diseases such as spinal muscular atrophy and Duchenne muscular dystrophy.
RNA Therapy FAQs
Q1: What is RNA used for?
Ans: It is used for carrying biological information, providing structure, facilitating chemical reactions and regulating the functions of DNA and other RNA molecules.
Q2: What does RNA stand for in therapy?
Ans: RNA stands for ribonucleic acid and it is a similar molecule to DNA.
Q3: What is an example of RNA therapy?
Ans: antisense oligonucleotide (ASO), small interfering RNA (siRNA), and microRNA (miRNA) therapies.
Q4: What are the benefits of RNA?
Ans: beneficial for injury recovery, post-surgery healing, memory support, digestive health, and managing the visible signs of aging.
Source: TH