Huntington's disease successfully treated in vitro for the first time using CRISPR gene editing

1.What is Huntington's disease?

Huntington's disease is an inherited disorder that results in death of brain cells. The earliest symptoms are often subtle problems with mood or mental abilities. A general lack of coordination and an unsteady gait often follow.

As the disease advances, uncoordinated, jerky body movements become more apparent. Physical abilities gradually worsen until coordinated movement becomes difficult and the person is unable to talk. Mental abilities generally decline into dementia. Full-time care is required in the later stages of the disease.

Symptoms usually begin between 30 and 50 years of age, but can start at any age. The disease may develop earlier in life in each successive generation. About 8% of cases start before the age of 20 years and typically present with symptoms more similar to Parkinson's disease.

People with Huntington's disease often underestimate the degree of their problems. Complications such as pneumonia, heart disease, and physical injury from falls reduce life expectancy. Suicide is the cause of death in about 9% of cases. Death typically occurs fifteen to twenty years from when the disease was first detected.

Huntington's disease affects about 4 to 15 in 100,000 people of European descent. The disease affects men and women equally.

2.Can Huntington's disease be cured?

There is no cure for Huntington's disease, yet.

However, researchers have shown promising results for a potential cure for Huntington’s disease. Using the genetic therapy known as CRISPR, the team was able to provide a permanent therapeutic treatment for this condition in mice.

The team used CRISPR to change the genes of the mice. The genetic therapy was delivered to the mice's brain cells using an adeno-associated virus. The viral carrier was injected in the brain striatum of the mice, the region that controls movement.

The study revealed the capacity of brain cells to heal themselves if the genetic source of the toxic proteins is removed. Moreover, in comparison with control Huntington's mice, CRISPR-injected mice showed significant improvements on tests of motor control, balance, and grip strength.

These findings open up an avenue for treating Huntington's as well as other inherited neurodegenerative diseases. The researchers showed that the gene mutations caused by CRISPR in this setup happened only in the Huntington genes and not in off-target genes.

Yet, there are currently no human clinical trials underway using CRISPR to fight neurodegenerative disorders like Huntington's disease, the opposite is the case for using CIRSPR to cure cancer and HPV (see https://steemit.com/science/@meisi51/hpv-about-to-be-cured-by-crispr-very-soon).

The potential for CRISPR to be a game-changing weapon in the medical arsenal is undeniable.

For further information:
http://www.iflscience.com/health-and-medicine/scientists-reverse-huntingtons-disease-in-mice-using-crispr/

http://www.genengnews.com/gen-news-highlights/crispr-reverses-huntingtons-disease-in-mice/81254532

https://www.sciencealert.com/crispr-could-point-to-a-cure-for-huntington-s-disease-suggests-new-study