A new study from the University of Minnesota is the first to demonstrate the ability for gene therapy to repair neural connections for those with the rare genetic brain disorder known as Hurler syndrome. The findings suggest the use of gene therapies—an entirely new standard for treatment—for those with brain disorders like Hurler syndrome, which have a devastating impact on those affected.
are dangerous and lifetime enzyme replacement fails to prevent progressive brain damage
The investigators observed the new approach of PS gene-editing produced normal enzymes from the liver that were able to sustain normal connections within specific neural networks. The technology needed for high resolution rs-fMRI brain connectome imaging was developed by Wei Zhu, a graduate student in the University's Center for Magnetic Resonance Research.
"A similar rs-fMRI approach as applied in this preclinical study should be translatable to the clinical setting and patients, especially for those with genetic, and for examining the efficacy of brain network restoration and function after gene treatment," said Wei Chen, a co-senior author and professor in the U of M Medical School and Center for Magnetic Resonance Research.
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