Gene therapy attempts to use to prevent Alzheimer's disease


2019-03-03 12:45:15




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Gene therapy attempts to use to prevent Alzheimer's disease

No One knows for sure what causes Alzheimer's disease. But one fact about this disease acquired almost irrefutable status. Depending on which version of the APOE gene you inherit, the risk of brain disease may be two times below the average — or 12 times higher. APOE is sometimes called the "genome of forgetfulness", and he gets three versions: 2, 3 and 4. Version 2 reduces the risk to humans; 3 — average; 4 — increases the risk drastically.

The Risk is so great that doctors avoid inspection of APOE because a bad result may upset the person and there's nothing you can do about it. There is no cure, and the genes cannot be changed.

Can you?


is there a cure for Alzheimer's genetically?

Not Yet. But doctors from new York say that starting in may, they will begin testing a new gene therapy in which people with the bad genes APOE will give a huge dose version of reducing risk.

If it helps to slow slow the depletion of brain disease in people who already have Alzheimer's disease, ultimately it will lead to the possibility of preventing the disease. Clinical trials, led by Ronald crystal of Weill Cornell Medicine in Manhattan, represents a new tactic against dementia, as well as a new twist in gene therapy. Most efforts to replace genes that rely on viruses that carry the DNA's instructions in human cells aimed at addressing rare diseases such as hemophilia, by replacing a single faulty gene.

But common diseases such there is no single reason why gene therapy has never been particularly promising. Trade group Alliance for Regenerative Medicine asserts that currently no gene therapies not performed on patients with Alzheimer's.

"it Seems that the path to clinical trials in humans will be long, but there is an urgent need for any treatment," says Kieran Musunuru, Professor of the medical school of the University of Pennsylvania. He studies genetic methods of treatment of cardiovascular diseases and says that the experiment, scheduled in new York, represents a new category of gene therapy, which aim is not to cure, but to reduce the risk of future disease in healthy people.

The crystal says his plan also bypasses the debate about the true cause of Alzheimer's disease, which has become a field of dreams multi-billion dollar, which lose and pharmaceutical companies, and patients. In January, Roche stopped two large studies of antibodies, which were supposed to clarify the properties of beta-amyloid plaques, the latter of theories, which claimed that these plaques around the neurons lead to the emergence of Alzheimer's.

"In the field there are many who firmly believe that the blame for amyloid," says crystal. Others believe that the blame for other protein Tau tangles which are found in the dying neurons. "The answer is likely to be difficult to find. The approach we have chosen, ignores all this and sees the situation from a genetic point of view."

The team of Crystal relies on 25 years of discovery. In the 1990's, scientists from Duke University were in search of proteins that could be attached to the amyloid plaques. And they have identified apolipoprotein-e, which is encoded APOE gene. Sequeira this gene in 121 patients, they found that a separate version — APOE4 — has inexplicably been distributed to those suffering from the disease.

The Function of this gene is still not well understood (it plays a role in the transport of cholesterol and fats), but its status as a risk factor remains intimidating. According to the Alzheimer's Association, approximately 65% of people with Alzheimer's have at least one copy of the dangerous gene. For people born with two copies of the high risk, one from each parent, dementia is almost guaranteed, if they live long enough.

However, some people inherit one 4 and one 2, version of the gene with low risk. These people are closer to the average risk, suggesting that the protective version of the gene compensates for the risk.

This effect the doctors at Weill Cornell I try to copy. Currently, the center is looking for people with two copies of the gene at high risk, who have lost memory or even got Alzheimer's. According to Crystal, about a month later the first volunteers will receive an infusion into the spinal cord of the billion viruses carrying gene 2.

Based on tests on monkeys, crystal expects that the virus will spread the "happy gene" in cells throughout the brain of the patient. Mice were treated similarly, and rodents accumulated less amyloid in the brain.

This strategy, according to the researcher, does not depend on knowledge about what really causes the disease. "In Alzheimer's we're attracted to evidence of genetic epidemiology," he says. "So the strategy is: can we atone for the brain to E2? We have the infrastructure, so we thought, why not? This solves the problem of the mechanism of disease."

"the Concept of rational" adds crystal. "If it works with the person is another question."

The new York study will be preliminary. Crystal said that his team needs to determine functioning and the added gene at the level at which it can be detected. The doctors take patients ' cerebrospinal fluid and check whether it contains the expected mix of proteins — the expected type 4 but now with equal to or greaternumber 2 is involved.

By the time when people start to forget names and where the car keys, changes in the brain are already a dozen years. This means that patients who joined the study, can not count on much. For them it will be too late.

Despite this, the Fund finding a cure for Alzheimer's gave Crystal $ 3 million on research. In the end, the hope is that middle-aged people with the genes in the risk zone can be a single genetic set up. Even a small reduction in the speed with which changes occur in the brain, may eventually change the situation.

Well, let's hope that everything will work out. We offer you to follow the news .


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