Scientists Uncover New Organelle in Cells That May Hold Key to Treatments for Genetic Diseases

Researchers at the University of Virginia School of Medicine and the National Institutes of Health have discovered a new organelle inside human cells, dubbed “hemifusome.” This specialized structure plays a crucial role in sorting, recycling, and discarding essential cargo within cells. The discovery could lead to new treatments for devastating genetic disorders.

The hemifusome helps manage how cells package and process material, with problems contributing to diseases such as Hermansky-Pudlak syndrome, which affects vision, lung function, blood clotting, and more. According to Dr. Seham Ebrahim, the discovery of this organelle is like finding a new recycling center inside the cell.

The research team used advanced imaging techniques to visualize the hemifusome, revealing that it facilitates the formation of vesicles – tiny sacs that act as mixing bowls – and organelles made up of multiple vesicles. This process is critical for cellular sorting, recycling, and debris disposal.

Hemifusomes are surprisingly common in certain parts of our cells and may be contributing to disease when they malfunction. Further research could lead to targeted treatments for complex genetic disorders, offering new hope for patients suffering from these devastating conditions.
Source: https://phys.org/news/2025-06-scientists-unknown-organelle-cells.html