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 gene-editing therapies, RNA-based therapies offer a safer alternative as they make temporary changes that do not 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.
