Gene Therapy to Strike Neurogenerative Huntington's Disease

According to French researchers, tested gene therapy on lab animals has showed promise in fighting the tragic neurogenerative disease known as Huntington's.

Gene therapy is one of the most exciting fields of medical research. Gene therapy to offer the tantalizing prospect of being able to slow, stop or even reverse a disease by correcting a flawed gene.

Scientists and researchers at the Institute of Biomedical Imaging and Molecular Imaging Research Centre (part of France's Atomic Energy Commission [CEA]) were experimenting with a modified virus to deliver a corrective gene into brain cells that boosts a natural shield against huntingtin's effects.

The symptoms of Huntington’s are jerky movement, uncontrolled, leading to personality change, dementia and death, which occurs between 10 and 20 years after the first signs emerge. Huntington’s disease strikes around one person in 10,000, with the problems mainly showing up between the early 30s and the age of 50.

A flaw in a single gene on Chromosome 4 called IT15 causes Huntington’s. Moreover, it causes a mutated form of a protein called huntingtin to run riot, killing cells in the striatum region of the brain. The neuroprotective molecule is called ciliary neurotrophic factor (CNTF).

According to Nicole Deglon, a French researcher, when the brain has a lesion or a problem, then the synthesis of CNTF is increased. Like a Trojan horse, the virus is injected into the striatum, where it infects brain cells, inserting the gene for CNTF.

Those French researchers have experimented on lab rats, followed by primates. The results showed that the technique was able to protect striatum cells. Recently, the team is moving towards a clinical trial on humans. The result of research was unveiled at a five-day conference in Geneva, organized by the Federation of European Neuroscience Societies (FENS).

Nevertheless, U.S. researchers at Rush University Medical Center, Chicago, has also worked with the similar project in gene therapy area using a defective cold virus to deliver a different gene, called glial-derived neurotrophic factor (GDNF), into mice brains. In addition, GDNF controls a protein that protects brain cells, which are at threat from Huntington's.

Meanwhile, enthusiasm has also been dampened by several bad setbacks when experiments that did well on lab animals backfired when tested on humans. Clinical trials are long because they are hedged with conditions and periodic reviews to ensure the prototype treatment is safe. In addition, British researchers in the latest issue of the Journal of Experimental Medicine said they had found that an inflammation-causing protein called IL-6 could serve as an early warning sign of Huntington's. Moreover, people with high levels of this protein went on to develop symptoms of the disease more than a decade later.

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