"Can Oligonucleotides Target RNA Splicing to Treat Disease?" from OTS

[JonMoulton]

TL;DR Summary

Nice introduction to splice-modification therapy from the Oligonucleotide Therapeutics Society

From the Oligonucleotide Therapeutics Society: "Can Oligonucleotides Target RNA Splicing to Treat Disease?"
https://www.oligotherapeutics.org/can-oligonucleotides-target-rna-splicing-to-treat-disease/

 

[Greg Bernhardt]

Yes, it appears that oligonucleotides can target RNA splicing to treat disease. In fact, there is a growing body of evidence that suggests that this type of therapy may be useful in treating a wide range of diseases, including cancer and genetic disorders. Oligonucleotides have already been used to target specific genetic mutations in some cancers, and they are being further developed to target other types of genetic mutations as well. Additionally, researchers are exploring ways to use oligonucleotides to modulate the expression of genes involved in various diseases, including metabolic and neurological disorders. Therefore, it appears that oligonucleotides have great potential for targeting RNA splicing in order to treat a variety of diseases.