Reimagining Drugs to Treat Rare Brain Disorder
In a recent breakthrough, researchers from the University of California have successfully used a new approach to treat a rare brain disorder. The team developed a new drug, which has potential to be effective in treating a rare genetic disorder called X-linked adrenoleukodystrophy (X-ALD). X-ALD affects the brain and the nervous system and is caused by an inherited mutation in a gene called ABCD1.
This new drug works by re-engineering the ABCD1 gene, replacing it with a healthy, functioning gene. This approach is called gene therapy and has the potential to revolutionize the treatment of X-ALD and other rare genetic diseases. The team was able to show that the new drug was effective in reducing the neurological damage associated with X-ALD in mice. This is a promising step forward in our understanding of how to treat this rare disorder.
source: Phys.org