CRISPR screening identifies TRIM27 as a destabilizer of the Smith–Magenis syndrome protein RAI1 – published May 16, 2026

Researchers wanted to better understand how the body controls RAI1 protein levels inside cells. They discovered that another protein, called TRIM27, helps break down RAI1. In SMS, where individuals already have reduced RAI1 levels, this breakdown may make the problem worse. Importantly, the researchers found that reducing TRIM27 activity in SMS mouse neurons partially improved some of the cellular features associated with the disorder. This suggests that slowing down the breakdown of RAI1 could become a new therapeutic strategy for SMS. In simple terms, instead of replacing the missing gene, scientists may also be able to help the remaining healthy copy of RAI1 work better and last longer.

Here is a link to the full research article: academic.oup.com