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[ Proteins Behaving Badly Provide Insights For Treatments Of Brain Diseases ]

Proteins Behaving Badly Provide Insights For Treatments Of Brain Diseases

A research team led by the University of Melbourne has developed a novel technique that tracks diseased proteins behaving badly by forming clusters in brain diseases such as Huntington's and Alzheimer's. The technique published in Nature Methods is the first of its kind to rapidly identify and track the location of diseased proteins inside cells and could provide insights into improved treatments for brain diseases and others such as cancer. Developed by Dr Danny Hatters and his team of the Department of Biochemistry and Molecular Biology at the Bio21 Institute, University of Melbourne, the technique uses a flow cytometer to track the protein clusters in cells at a rate of 1000s per minute. In addition, cells with clustered proteins can be recovered for further study - neither of which had been possible before.

Huntington's Disease - Stem Cell Therapy Potential

At present there is no effective treatment for Huntington's disease - a progressive disorder in which nerve cells in certain parts of the brain waste away or degenerate and affects muscle coordination. However, according to a study published March 15 in the journal Cell Stem Cell, a special type of brain cell created from stem cells could help restore the muscle coordination deficits that are responsible for uncontrollable spasms, a characteristic of the disease. The researchers demonstrated that movement in mice with a Huntington's-like condition could be restored. Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the study, said: "This is really something unexpected." In the study Zhang, who is an expert in creating various types of brain cells from human embryonic or induce pluripotent stem cells, and his team focused on GABA neurons.

Stem Cells Hint At Potential Treatment For Huntington's Disease

Huntington's disease, the debilitating congenital neurological disorder that progressively robs patients of muscle coordination and cognitive ability, is a condition without effective treatment, a slow death sentence. But if researchers can build on new research reported this week (March 15, 2012) in the journal Cell Stem Cell, a special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of the disease. "This is really something unexpected, " says Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition. Zhang is an expert at making different types of brain cells from human embryonic or induced pluripotent stem cells.

Widely Held View On Causal Mechanism In ALS Being Questioned

In science, refuting a hypothesis can be as significant as proving one, all the more so in research aimed at elucidating how diseases proceed with a view toward preventing, treating, or curing them. Such a discovery can save scientists from spending precious years of effort exploring a dead end. In a study published in the Proceedings of the National Academy of Sciences, Munich-based researchers refute a widely accepted hypothesis about a causative step in neurodegenerative conditions. These results deal specifically with animal models of human amyotrophic lateral sclerosis (ALS, aka Lou Gehrig's disease ) but also raise questions for research on other neurodegenerative diseases, such as Alzheimer's or Huntington's disease. One of the ways neurodegenerative diseases manifest themselves is in the loss of axons - essentially, the transmission lines for electrical signals in individual nerve cells - and synapses, the key sites for communication between them.

Previously Unknown Mechanisms In The Pharmacology Of Dopamine Stabilisers

A study from Karolinska Institutet shows that a new drug for Huntington's disease - pridopidine or dopamine stabiliser ACR16 - might operate via previously unknown mechanisms of action. Researchers have found that at very low concentrations, ACR16 binds to the sigma-1 receptor, a protein in the brain important to neuronal function and survival. This new knowledge can be used to develop future treatments for schizophrenia, involuntary Parkinsonian tremors and neurodegenerative diseases. "It's conceivable that some of the beneficial effects of dopamine stabilisers are mediated via the sigma-1 receptor, " says principal investigator Daniel Marcellino of the Department of Neuroscience. "Our results suggest a formerly overlooked aspect of dopamine stabiliser pharmacology." Dopamine stabilisers are a new class of drug substance originally developed by Swedish Nobel laureate Professor Arvid Carlsson.

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