PS gene-editing shown to restore neural connections lost in brain disorder
MINNEAPOLIS/ST. PAUL (08/21/2023) — 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.
The study was published in the Nature journal Scientific Reports.
Hurler syndrome, also known as mucopolysaccharidosis type I (MPS I), is a genetic disorder affecting newborns ...





