RNA Therapy

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.

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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