French researchers say that gene therapy showed great promise in combating the tragic neurogenerative disease: Huntington’s Disease.
The Process Behind Huntington’s Disease
The disease strikes around one person in 10,000, with the problems mainly showing up between the early 30s and the age of 50. Its symptoms are uncontrolled, jerky movement, leading to personality change, dementia and death, which occurs around 15 years after the first signs emerge. Huntington’s is caused by a flaw in a single gene (IT15) on Chromosome 4. This genetic defect causes a mutated form of a protein called huntingtin to kill cells in the brain. It is a dominant trait, so if one parent has it, offspring have a 50% chance of getting the disease, and if both have it, the child will certainly get the problem gene.
Scientists at the Institute of Biomedical Imaging and Molecular Imaging Research Centre were experimenting with a modified virus to deliver a corrective gene into brain cells that boosts a natural shield against the problems caused by huntingtin’s. The protection comes in the form of a ciliary neurotrophic factor, or CNTF. When the brain is cut or smashed, the synthesis of CNTF is increased as it heps neurons to survive the onslaught. Like a Trojan horse, the virus is injected into the affected part of the brain, where it infects cells, inserting the gene for CNTF.
Experiments on lab rats, followed by primates, show that the technique does protect striatum cells, and the team is now moving towards a clinical trial on humans. Other genes, like the glial-derived neurotrophic factor have been inserted into brain tissue as well via virus, and produced similar effects.
Gene therapy shows prospect of being able to slow, stop or even reverse a disease by correcting a flawed gene. However, many experiments that did well on lab animals performed poorly on humans.
British researchers have also said that they had found that an inflammation-causing protein called IL-6 could serve as an early warning sign of Huntington’s. People with high levels of this protein went on to develop symptoms of the disease more than a decade later. This early warning system, combined with the new gene therapy, might help combat Huntington’s.