Huntington's Disease: Research Shows Gene Therapy to be Effective Combatant

French researchers have announced that gene therapy that has been tested on subjected lab animals shows promising results for combating the tragic neurogenerative disease know as Huntington's disease. The disease is known to strike nearly 1 in every 10,000 people with the problems from the disease starting to show in the 30 - 50 age range. It's symptoms are as follows: Jerky uncontrolled movement, personality change, dementia and ultimately death. Death of the individual usually occurs between 10 and 20 years after the first initial signs of the disease. Huntington's disease is caused by a flaw in a single gene on Chromosome 4 called IT, it causes a mutated form of a protein called Huntington to run riot and it begins killing cells in the striatum region of the brain. Scientists said they were experimenting with a modified virus to deliver a corrective gene into brain cells that boosts a natural resistance against the Huntington's disease effects.

The neuroprotective molecule is called ciliary neurotropic factor, or CNTF for short. Basically what will happen is when the brain is having a problem or a lesion, then the synthesis of CNTF will be increased to better help the neurons survive. The virus is injected into the striatum, where it infects brain cells, inserting the gene for CNTF. They conducted experiments on lab rats, followed by primates to show that the technique does in fact protect the striatum cells, now the team of scientists are now moving on to clinical trials on humans.

Their hope is that the patient will benefit for several more years through the testing. Their research was revealed mid July at a five day conference in Geneva, organized by the Federation of European Neuroscience Societies. There is also similar work in this field that is being carried out by US researchers at Rush University Medical Center in Chicago. Using a defective cold virus to deliver a different gene known as glial-dervied neurotrophic factor into the brains of mice. Glial-derived neurotropic factor controls a protein that protects brain cells that are at threat from Huntington's.