There’s more great news coming towards us from the medical space. Check out the latest reports about the Hurler Syndrome.
Hurler Syndrome breakthrough
The University of Minnesota conducted a groundbreaking study that proved gene therapy can restore neural connections in individuals with Hurler syndrome, a rare genetic brain disorder. This discovery could pave the way for gene therapies to become the new standard of treatment for brain disorders, such as Hurler syndrome, that cause immense suffering to those affected.
The study was published in the Nature journal Scientific Reports.
Hurler syndrome, also referred to as mucopolysaccharidosis type I (MPS I), is a genetic condition that affects newborns and causes severe cognitive impairments and physical abnormalities.
The disorder is caused by genetic mutations that hinder the production of a vital lysosomal enzyme called IDUA, which leads to progressive brain damage and ultimately, death before the age of 10. Existing treatments are insufficient, with bone marrow transplants being risky and enzyme replacement therapy failing to halt the progression of brain damage.
Testing a new gene therapy
Researchers at the University of Minnesota have tested a new gene therapy called the PS gene-editing system on mice with Hurler syndrome. The therapy resulted in the production of healthy enzymes in the liver, which could enter the brain through the circulatory system.
To evaluate the effectiveness of the therapy, the researchers used high-resolution resting-state functional MRI (rs-fMRI), which is a safe and noninvasive imaging tool that can assess brain activity and connectivity.
The researchers first identified disrupted neural networks and then measured the restoration of brain functions and connectivity following the gene therapy.
Walter Low, a co-senior author and professor in the U of M Medical School, said that this study is a breakthrough: “This is the first demonstration of a gene therapy that has corrected a neurological disorder resulting in the restoration of brain connectivity as confirmed by rs-fMRI.”
“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 brain disorders, 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.