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Recently, researchers have prevented the development of Alzheimer's disease in mice by using a virus to introduce a specific gene into the brain. The study was funded by the UK Research Center for Alzheimer's Disease and the European Research Council, and the results were published in the October 10 issue of PNAS.
These early discoveries, led by scientists at Imperial College, have opened up a new path to potential new treatments for the disease. In this study, the team used an improved virus to pass a gene, called PGC1-α, to brain cells. Previous studies led by the same team have shown that this gene prevents the formation of a beta-amyloid in cells in the laboratory.
--amyloid is a major component of amyloid plaques and is a sticky mass of protein found in the brains of Alzheimer's patients. These plaques are thought to trigger the death of brain cells.
In the UK, Alzheimer's disease affects approximately 520,000 people. Symptoms of the disease include memory loss, confusion, and changes in mood or personality. Worldwide, 47.5 million people are affected by dementia, with Alzheimer's disease being the most common form. There is currently no cure for Alzheimer's disease, and current drugs can only help alleviate the symptoms of the disease.
Dr. Magdalena Sastre, a senior author of the paper and a professor of medicine at Imperial College, said that it is hoped that these new discoveries will provide a way to prevent the disease or stop it at an early stage. She explained: "Although the results of these studies are too early, they point out that this gene therapy may have potential therapeutic uses for patients. There are still many obstacles to overcome. The only way to do this is by direct injection of the gene. Passed into the brain. However, this proof-of-concept study suggests that this approach requires further investigation."
Professor Nicholas Mazarakis, co-author and medical department of the study, explained: “The improved virus used in the experiment is called lentiviral vector and is widely used in gene therapy. Scientists use a way of infecting cells with lentivirus to produce a modified version. The virus, which is introduced into specific cells. It is used in experiments to treat a range of diseases from arthritis to cancer. We have successfully used lentiviral vectors in clinical trials to transfer genes to Parkinson's disease. In the patient's brain."
In this new study, the team injected a virus containing the gene PGC-1α into two brain regions of mice susceptible to Alzheimer's disease. The targeted regions are the hippocampus and cortex, as these regions are the first regions of Alzheimer's disease that form amyloid plaques.
Damage to the hippocampus can affect short-term memory and cause one to forget recent events, such as a conversation or what breakfast they ate. The hippocampus is also responsible for positioning, and its damage can lead to a person not knowing the route that was once familiar, such as driving home from the store. At the same time, the cortex is responsible for long-term memory, reasoning, thinking and emotions. Cortical damage can cause depressive symptoms, such as depression, trying to figure out how much to pay at checkout, how to dress or how to cook a familiar dish.
Animals were treated in the early stages of Alzheimer's disease, when they still did not develop amyloid plaques. Four months later, the team found that mice receiving the gene had very few amyloid plaques compared to untreated mice, with multiple plaques in the brain.
In addition, treated mice performed the same as healthy mice in memory tasks. The task includes some challenges, such as replacing a familiar object in a mouse cage with a new one. If mice have a healthy memory, they will explore new objects for a long time.
The team also found no brain cell loss in the hippocampus of mice treated with gene therapy. In addition, the number of glial cells in the treated mice is reduced, and in Alzheimer's disease such cells may release toxic inflammatory substances, leading to further cellular damage.
The protein PGC-1α is encoded by the PGC-1α gene and is involved in metabolic processes in the body, including regulation of sugar and fat metabolism. Dr. Sastre added that other studies from different institutions have shown that physical exercise and compound resveratrol, which are present in red wine, may increase the level of PGC-1α protein. However, studies have found that resveratrol is only beneficial when taken as a tablet, rather than from alcohol, because alcohol can offset any benefits it brings. The team recommends that the injection of this gene is most beneficial in the early stages of the disease, when the first symptom occurs.
Dr. Sastre said that now they want to explore the transformation of their research into human therapy. “We still need years to move to clinical use. However, for a disease that urgently needs new options, this work provides hope for future therapies.â€
Dr. David Reynolds, Chief Scientific Officer of the Alzheimer's Disease Research Centre in the United Kingdom, said: "There is currently no treatment to prevent the progression of Alzheimer's disease, so research like this is very important and it is hoped to find new and innovative ways to make us Close to this goal. This study laid the Foundation for 'exploring gene therapy as a treatment strategy for Alzheimer's disease', but further research is needed to determine whether gene therapy is safe and effective for patients with such diseases. And practical. These findings support PGC-1-α as a potential target for the development of new drugs, a welcome step on the road to developing treatments for this devastating disease."
Exploring gene therapy to break through or hope to cure Alzheimer's disease
[China Pharmaceutical Network Technology News] In the UK, Alzheimer's disease affects about 520,000 people. Symptoms of the disease include memory loss, confusion, and changes in mood or personality. Worldwide, 47.5 million people are affected by dementia, with Alzheimer's disease being the most common form. There is currently no cure for Alzheimer's disease, and current drugs can only help alleviate the symptoms of the disease.