Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies
Peer-Reviewed Publication
Updates every hour. Last Updated: 11-Jul-2025 19:10 ET (11-Jul-2025 23:10 GMT/UTC)
JAX scientists have developed a mouse models for AHC, a rare and severe neurological disorder in children, paving the way for gene-editing therapies and deeper understanding of disease progression and treatment.
Chronic inflammation occurs when the immune system is stuck in attack-mode, sending cell after cell to defend and repair the body for months or even years. Diseases associated with chronic inflammation, like arthritis or cancer or autoimmune disorders, weigh heavily on human health—and experts anticipate their incidence is on the rise. A new study by investigators from Mass General Brigham identified a protein called WSTF that could be targeted to block chronic inflammation. Crucially, this strategy would not interfere with acute inflammation, allowing the immune system to continue responding appropriately to short-term threats, such as viral or bacterial infection. Results are published in Nature.