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Scientists develop a Drug likely to benefit people with Alzheimer’s Disease

FILE - An image shows activity in a human brain. Scientists have developed a drug capable of sweeping away abnormal protein clumps in the brain which are a hallmark of Alzheimer’s disease. VOA

Washington, Jan 27, 2017: Scientists have developed a drug they hope will benefit people with Alzheimer’s disease, which afflicts an estimated 44 million people around the world. The new compound sweeps away abnormal protein clumps in the brain which are a hallmark of the neurodegenerative disorder.

In a study reported in the journal Science Translational Medicine, researchers describe how a synthetic drug, called antisense oligoneucleotide, reduced the production and in some cases cleared clumps of tau in the brain.

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Tau bundles are one of the hallmarks of the disease, along with beta amyloid deposits, another destructive protein.

By stopping the formation of tau, researchers at Washington University School of Medicine in St. Louis, Missouri, found they could extend the lives of mice that were bred to have collections of human tau in their brains.

Lead author Sara DeVos said scientists saw an improvement in their condition.

“So these mice die earlier than normal. So when we treat with our drug, the mice live longer and we can also prevent neurons from dying. So if we give this drug, the neurons will no longer die as a result of these tau bundles,” said DeVos

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The investigators also tested the compound in monkeys and saw positive results.

FILE – Patients with Alzheimer’s and dementia are seen during a therapy session. Alzheimer’s afflicts an estimated 44 million people around the world. VOA

Human testing expected soon

Antisense oligneucleotide targets the genetic instructions for building tau. The molecule binds to messenger RNA, which carries out the DNA blueprint for life, preventing tau from being produced. The drug can be made to target RNA for destruction of any protein, said scientists.

Tim Miller, a professor of neurology at Washington University and senior author of the study, hopes the drug, developed with Ionis Pharmaceuticals, will soon be tested in humans with Alzheimer’s disease.

“The most exciting and most interesting … is to apply this to people who we presume have abnormal tau to test the hypothesis whether lowering tau in those people will be of benefit to those people,” he said.

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Other types of antisense oligoneucleotides have been approved by U.S. regulators and are being used to treat the neurodegenerative disease muscular dystrophy and spinal muscular atrophy. The compound is in clinical trials for Huntington’s disease and ALS, also known as Lou Gehrig’s disease.

Because tau deposits are only a piece of the puzzle that causes Alzheimer’s, the investigators envision using the drug with other treatments, also in development.

There is no cure for Alzheimer’s disease, which primarily strikes senior adults, leading to a decline in mental functioning and eventually death. (VOA)

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Scientists identify 36 new genes implicated in cardiac disease

They found the transcription factor gene was directly connected to a whole network of proteins known to play a role in cardiac hypertrophy

Scientists discovered 36 new genes related to cardiac diseases. IANS
Scientists discovered 36 new genes related to cardiac diseases. IANS
  • Scientists discovered 36 new genes
  • These are the genes implicated in cardiac diseases
  • These genes play a casual but important role in the diseases of heart

Researchers have identified 36 new genes that plays a causal role in cardiac hypertrophy — abnormal thickening of the heart muscle — which can lead to heart failure.

These genes can cause heart failure. Pixabay
These genes can cause heart failure. Pixabay

“This is an exciting direction for personalised medicine, and also for identifying genes and therapeutic targets for complex diseases that involve many genes,” said co-author of the study Alain Karma, professor at the Northeastern University. According to the Centres for Disease Control and Prevention, one in four deaths in the US each year are due to heart disease.

Also Read: Heart Surgery In Infants May Cause Deafness

“The framework described in the paper can also be used to predict whether individuals suffering from a particular disease will respond to a given drug treatment,” said lead author Marc Santolini, a postdoctoral research associate at Centre for Complex Network Research.

For the study, published in the journal npj Systems Biology, the researchers took an entirely different approach — using the Hybrid Mouse Diversity Panel, a collection of 100 genetically different strains of mice that can be used to analyse the genetic and environmental factors underlying complex traits.

The Science of Genetics behind Gotra system
The study was done on many kinds of genes and chemicals, Wikimedia Commons

Within each strain, the mice are inbred, making them all identical twins on a genetic level. Researchers took two mice from the same strain and gave one of them a stressor drug that induces heart failure. They then compared the stressed mouse’s gene expression with its non-stressed twin.

Since the mice have the same genome, they were able to pinpoint individual genes that changed expression as a direct result of the heart stressor.

Also Read: Heart Attack Symptoms In Women Often Misinterpreted

Among the identified 36 such genes, many of these genes were previously unknown to be implicated in heart failure, the researcher said. They mentioned that one of them is known as a transcription factor, meaning it controls the expression of many other genes.

They confirmed the gene’s role by using molecular biology techniques to silence it and observe the resulting changes of expression.

blood type
It is important to take care of one’s heart to avoid major health issues. Pixabay

They found the transcription factor gene was directly connected to a whole network of proteins known to play a role in cardiac hypertrophy. IANS