Research

To understand ASO therapy, you first need to grasp how our cells make proteins. It all starts in the nucleus with DNA, our permanent "recipe book." When a cell needs a protein, it doesn't read the DNA directly. It makes a temporary copy called messenger RNA (mRNA), like photocopying a recipe to take to the kitchen.

In recent years, ASO (antisense oligonucleotide) therapies have been transforming the landscape of rare genetic diseases. Several treatments have been approved and dozens more are in development. Although we don't yet know whether this approach will be applicable to the LMBRD2 gene, it's important to understand these advances and their implications.