Rewiring Decline: Targeting Early Neurodegeneration in Sanfilippo Syndrome Through Gene Correction and Molecular Parallels with Alzheimer’s Disease

Sanfilippo syndrome (MPS III) is a rare but very serious neurodegenerative disorder that affects children. It happens when the body can't break down a molecule called heparan sulfate, which then builds up in the brain and causes inflammation, damage, and shrinkage over time. Most kids look healthy when they're born, but between ages two and six, they start losing speech and motor skills, and the diagnosis usually comes too late. This project focuses on figuring out what happens early in the brain, fixing the genetic glitch that causes it, and finding a way to get that fix where it actually works.