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Scientists Reverse Huntington’s Disease In Mice Using CRISPR

CI photos/Shutterstock

CI photos/Shutterstock

Researchers have made some initial but promising achievements in a potential treatment of Huntington’s disease. With the help of such genetic therapy named as CRISPR, the scientists were capable of offering a permanent therapeutic cure to this condition in mice.

Known as a fatal neurodegenerative disorder,  Huntington’s disease is caused by the breakdown of nerve cells in the brain. It’s an inherited condition that generally starts in adulthood and is caused by a gene that generates  proteins that are quite harmful to cells in the brain.

The recent research result , released in the Journal of Clinical Investigation, concentrated on mice engineered to develop Huntington’s disease, showing such symptoms as damaged movement developing when they are nine months old. By application of CRISPR, researchers  changed the genes of the mice. In the period of three weeks, the mice were significantly improved, although they were not back to the type of a normal mouse.

In this genetic therapy a virus was delivered to the mice’s brain. Such approach known as the adeno-associated virus, also called AAV, has been successful in CRISPR. The viral carrier was injected in the brain striatum of the mice, where movement.controls were made.

In a statement, Professor Xiao-Jiang Li, senior author from Emory University School of Medicine said that the discoveries had paved the way for treatment of Huntington’s as well as other inherited neurodegenerative diseases, however more testing in terms of safety and long-term effects were further required.

Talking of CRISPR medical researchers are more concerned about potential long-term effects. The potential for CRISPR as a phenomenal weapon in the medical treatment is unquestionable, but there’s still a long way to  go before scientists could fully understand it.

What would happen if another gene is affected when we suppress a specific gene known as the causing of a disease, ? Or what would occur if the change could cause  other long-term difficulties? The research result demonstrated that the gene mutations caused by CRISPR in this situation took place just in the Huntington genes and rather than off-target genes.

According to Li’s assessment, the long-term effects and safety of injecting AAV in the brain to express CRISPR/Cas9 needed to further tested before it could be actually applied to patients.

At present, application of CRISPR to fight neurodegenerative disorders in humans has not been planned, but human trials in making cells capable of fighting cancer are going on now in the USA and in China.