The original article 

https://www.nature.com/articles/s41392-020-0207-x

Conclusions

Due to the mechanism of action of siRNA, this molecule can be used to target nearly all disease-related genes of interest. The duration of drug research and development is also much shorter than that for small molecules, monoclonal antibodies, and proteins. After the successful commercialization of ONPATTRO® and GIVLAARI™ for the treatment of rare diseases,

(amyloidosis and porphyria) siRNA therapy will achieve another milestone soon for treating common diseases, e.g., dyslipidemia, as excellent phase 3 data have been collected from several clinical studies of inclisiran.

Benefiting from long-term and constant innovations in exploring modification geometries and delivery systems, the effective dose and half-life of the siRNA modalities were reduced from milligrams to 10−3 mg and prolonged from minutes to months, respectively. GalNAc–siRNA conjugates with sophisticated chemical modifications enable quarterly or twice-yearly subcutaneous administration of siRNA therapeutics, which is a great achievement that had never been realized in the history of pharmaceuticals. These improvements will exert widespread and far-reaching impacts on the whole medical industry, e.g., on drug development, government management, patient treatment, payment patterns of medical insurance, financial investments from the capital market, etc.

After studying GalNAc–siRNA conjugation for hepatic delivery of siRNA, researchers will devote themselves to developing extrahepatic delivery platforms. Several important advances have been achieved for renal, CNS, and ocular targeted delivery of siRNA. Therefore, state-of-the-art siRNA technologies will definitely change our lives in the future.

It appears that an exciting new era utilizing mRNA is indeed opening up.

By using the same technology of Pfizer and Moderna vaccines, specifically interfering messenger 

RNA(siRNA), we can now control the tough to treat familial hypercholesterolemia.

They are saying we may be able to do the same against many other diseases

including cancers. How exciting! I'm just awed by this development.

China and South Korea seem to be in the middle of it by reviewing the references.

Couldn't agree more, Dr. HJ. It is indeed an amazing technology we never thought of, even a few years back! 

So, there is another, non-viral vector to deliver m-RNA as a new way, Gene Silencing Treatment, instead of a traditional tool, using viral vectors! Fantastic!!!

It might change the whole concept of immunosuppressive therapy for all the varieties of organ/tissue transplantation as well. 

Lived long enough to see all these miracles(?), either good or bad, I don't know!

BB Lee

P.S. In my vascular malformation area, Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome, is the best-known condition to need genetic counseling for the inherited condition in an autosomal dominant manner. Though I had only two cases of AVMs caused by the HHT through the Johns Hopkins, one with GI involvement with such awful bleeding was helplessly losing game I recall. Such gene-silencing tech would be able to open a new chapter!!!