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Researchers Boost RNA Drug Effectiveness by Slowing Intracellular Transport

Life Sciences and Bio Pharma

Business Honor
01 July, 2025

Slower cell transport helps RNA drugs escape endosomes, improving treatment for genetic diseases.

Researchers at Roche and the University of Basel have discovered a method to improve the effectiveness of RNA-based medications which are used to treat uncommon genetic illnesses. These medications are called antisense oligonucleotides (ASOs) function prevents the production of toxic proteins by cells which are connected to hereditary disorders such as Duchenne muscular dystrophy and ALS.

Although ASOs are a promising treatment, their effectiveness is frequently subpar. This is because before they can reach their destination the majority of them become trapped inside the endosomes which are the cell's recycling centers. The medications are broken down and lose their effect once they are confined. In this study that was published in Nature Communications, researchers scanned hundreds of human genes using CRISPR gene-editing to identify those that influence the movement of ASOs within cells.

They found that ASOs have more time to get out of the endosomes and become active when their transport through the cell is slowed down. They discovered AP1M1 as an important gene that helps in the transfer of materials from endosomes to lysosomes which are responsible for the destruction of cell trash.

The ASOs had a better chance of escaping and functioning correctly when this gene was controlled because they remained in the endosomes longer. This approach significantly increased the drug's efficiency without raising the dosage according to tests conducted on mice and cells. Better treatments for various diseases may be developed as a result of this discovery which also provides new avenues for RNA therapy improvement. It also shows how important it is to follow cellular transport networks in order to improve the efficacy of life saving medications.