Gene Therapy Treats Rare Brain Disorder in Mice

Researchers from the University of Minnesota used gene therapy to help treat Hurler syndrome in mice. The results of this study can be used for further research on neurological disorders.

Hurler syndrome is a rare genetic brain disorder that affects newborns by causing severe physical abnormalities, cognitive deficiencies, and progressive brain damage. Due to those adverse effects of the disease, the patients experience death at the age of 10 years old.

Previously, there were no ideal treatments for people with Hurler syndrome. That's why the researchers decided to test adeno-associated virus (AAV)-mediated gene therapy in mice with Hurler syndrome. The team used resolution resting-state functional MRI (rs-fMRI) to identify neural connections that were lost. Next, they analyzed the effects of the gene therapy on brain functions and connectivity.

The results showed that gene therapy can reestablish the majority of brain connectivity. "The aeonic production of normal IDUA in the liver of mice with Hurler syndrome and the ability to traffic enzymes across the blood-brain barrier to correct abnormalities in the brain is a significant achievement," said Chester Whitley, a co-author of the study.

For more information, read the journal article on Scientific Reports.