RNA Therapy, Applications of RNA based Therapies

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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?