Researchers have shown preliminary but promising results for a potential remedy for Huntingtons disease. Utilizing the genetic therapy known as CRISPR, the group was able to provide a permanent therapeutic treatment with this condition in mice.
Huntingtons disease is a fatal neurodegenerative disorder which causes the breakdown of nerve cells in the brain. Its an inherited condition which typically begins inadulthood and is brought on by a gene thatproduces proteins thatare poisonous tocells in the brain.
The study, published in the Journal of Clinical Research, concentrated on mice engineered to develop Huntingtons disease, withsymptoms like impaired movement developing when they arenine months older. The group subsequently used CRISPR to modify the genes of their mice, and over three months, the mice had been significantly improved, but not back to the level of a healthy mouse.
The genetic treatment was delivered to the mice brain cells employing a virus. This approach called the adeno-associated virus, or AAV, has been successful in CRISPR. The viral carrier has been injected in the brain striatum of the mice, the area that controls motion.
The findings open up an avenue for treating Huntington’s and other bronchial diseases, although more testing of security and long-term effects is required, senior author Professor Xiao-Jiang Li, by Emory University School of Medicine, said in a statement.
The potential long-term effects are exactly what makes medical researchers tread carefully when it comes to CRISPR. The capacity for CRISPR to be a phenomenal weapon at the medical arsenal is incontrovertible, but theres still more that we have to understand.
Imagine if by curbing a specific gene we know is causing a disorder, a different receptor is affected? Or imagine if the change results in other long-term issues? The researchers showed that the gene mutations brought on by CRISPR in this setup happened just in the Huntington genes and not in off-target genes.
The long-term effects and safety of injecting AAV in the brain to state CRISPR/Cas9 remain to be rigorously tested before applying this approach to patients, Li added.
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