Huntington’s disease is a neurodegenerative disease that affects the brain cells.
There is a gene called HTT gene or the Huntingtin gene.This gene provides instructions for making a protein called huntingtin. Mutation in this gene causes Huntington’s disease. The mutated form of the HTT gene produces abnormal of the Huntington protein, which effects the brain cells. It is still not clear how this abnormal Huntingtin protein effects the brain cells.
Symptoms include involuntary movements known as chorea. It also affects a person’s thinking abilities, emotions and behavior.
POTENTIAL CURE??
An experimental drug was injected into the patients cerebro spinal fluid. This was done at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London. Basically, what this drug does is it prevents the HTT gene from creating the Huntingtin protein.
From the trials, it is known that there are no major safety concerns, side effects. And it is reducing the level of the Huntingtin protein as expected.
For patients whose conditions are already far advanced, the damage is already being done. So they will not benefit from this discovery. But for people who know that they are carrying the gene or are in the early stages, this discovery can really be a game changer for them as it could prevent the disease from progressing.
