Where should siRNAs go: applicable organs for siRNA drugs

Experimental & Molecular Medicine, Published online: 10 July 2023; doi:10.1038/s12276-023-00998-yRefining the design and delivery of RNA-based drugs could improve the chances of targeting diseases in complex organs. Gene expression is regulated by small interfering RNAs (siRNAs), which bind to messenger RNA sequences, preventing subsequent gene expression. Drugs based on siRNA show promise as safe and specialised for multiple diseases. Jinju Han and co-workers at the Korea Advanced Institute for Science and Technology in Daejeon, South Korea, reviewed the current status of siRNA drugs. Five siRNAs approved by the US Food and Drug Administration target the liver, while drugs targeting eye conditions and wound healing in the skin are in clinical trials. Targeting complex organs like the brain and lungs remains challenging, because the size of siRNAs and their delivery mechanisms must be adjusted in order to pass safely into target regions.
Source: Experimental and Molecular Medicine - Category: Molecular Biology Authors: Source Type: research