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Category: biotech/medical

Targeting enzyme could block cancer spread to brain with fewer side effects

A new study has identified a more precise and effective way to prevent cancer from spreading to the brain. The paper, published in the Proceedings of the National Academy of Sciences, details the development of novel drug candidates that target a key enzyme implicated in the spread of lung, breast, skin and other cancers to the brain. The work builds on a promising new therapeutic strategy first reported by the same group of researchers last year.

The new drug candidates are designed to intercept rogue cancer cells before they depart from primary tumors and ultimately travel to the brain.

Lead author Sheila Singh, based at both King’s College London and McMaster University, says this type of cancer—called metastatic brain cancer—is the most common type of brain tumor in adults and comes with an extremely grim outlook, with 90% of patients dying within one year of diagnosis.

UNM Researchers Find Alarmingly High Levels of Microplastics in Human Brains — and Concentrations are Growing Over Time

Microplastics – tiny bits of degraded polymers that are ubiquitous in our air, water and soil – have lodged themselves throughout the human body, including the liver, kidney, placenta and testes, over the past half century.

Now, University of New Mexico Health Sciences researchers have found microplastics in human brains, and at much higher concentrations than in other organs. Worse, the plastic accumulation appears to be growing over time, having increased by 50% over just the past eight years.

In a new study published in Nature Medicine, a team led by toxicologist Matthew Campen, PhD, Distinguished and Regents’ Professor in the UNM College of Pharmacy, reported that plastic concentrations in the brain appeared higher than in the liver or kidney, and higher than previous reports for placentas and testes.

Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates

Aeran and colleagues present research on targeted gene therapy vector engineering and pre-clinical testing of neuron-targeted AAV9-based constructs for STXBP1-related neurodevelopmental and epileptic encephalopathies. Candidate vectors designed to target specific neuronal types and detarget tissues associated with toxicity produced robust phenotypic reversal in Stxbp1 +/− mice and were well tolerated in monkeys.

Life Summit — Tomorrow.bio

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Lab-on-a-chip platform shows how immune cells attack cancer cells

Immunotherapies are a promising approach in the fight against cancer. Researchers at the Technical University of Munich (TUM) have developed a lab-on-a-chip system called CellTrap. It makes it possible to observe the interactions between immune cells and cancer cells at the single-cell level. The method is intended to reveal fundamental processes in cancer immunology and answer key questions. The technology is described in the journal RSC Advances.

Established laboratory tests mainly capture average values across many cells and show, for example, how many cancer cells survive after contact with immune cells. What happens in detail—how each cell reacts and interacts with others—remains hidden. However, to better understand the effectiveness of immunotherapies, the precise timing of a cell-cell interaction is often crucial: when contact, activation and, ultimately, the killing of the cancer cell occur.

Gene tied to energy production in brain could lead to new treatment for cognitive disorders

Researchers in the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo have discovered a connection between a specific gene and healthy brain function. “The hope is that this discovery could eventually lead to expanded treatment for psychiatric and neurological disorders such as schizophrenia, bipolar disorder and autism,” explains Mikhail V. Pletnikov, MD, Ph.D., professor and chair of the Department of Physiology and Biophysics, the senior author of the study with Kateryna (Kate) Murlanova, Ph.D., the first lead author and a research scientist in the department.

They discovered that the NPAS3 gene expressed in astrocytes—the cells that help with brain chemistry—regulates the energy production required to support thinking and memory. NPAS3 is a transcription factor, which means it directs how certain genes work and influences how cells function. Their findings are published in Science Advances.

“Previous studies have linked NPAS3 to conditions involving cognitive problems, such as schizophrenia, but scientists didn’t know exactly how it might be involved,” Pletnikov says.

The Role of Tau Pathology in Alzheimer’s Disease and Down Syndrome

Background: Individuals with Down syndrome (DS) exhibit an almost complete penetrance of Alzheimer’s disease (AD) pathology but are underrepresented in clinical trials for AD. The Tau protein is associated with microtubule function in the neuron and is crucial for normal axonal transport. In several different neurodegenerative disorders, Tau misfolding leads to hyper-phosphorylation of Tau (p-Tau), which may seed pathology to bystander cells and spread. This review is focused on current findings regarding p-Tau and its potential to seed pathology as a “prion-like” spreader. It also considers the consequences of p-Tau pathology leading to AD, particularly in individuals with Down syndrome. Methods: Scopus (SC) and PubMed (PM) were searched in English using keywords “tau AND seeding AND brain AND down syndrome”

Gene therapy shows promise in ARC syndrome, a deadly childhood liver disease

A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hospital. Arthrogryposis, renal dysfunction and cholestasis (ARC) syndrome is a lethal genetic disorder usually caused by a lack of the VPS33B protein, with children diagnosed with the condition rarely living beyond their first year of life.

Now, in a study published in Nature Communications, the UCL-GOSH team found that by injecting a healthy version of the gene into the body, they can treat the condition in mice lacking VPS33B. Crucially, the final version of the treatment, which specifically targeted the liver cells, caused no harm. In the earlier versions, the genes became abnormally activated and caused cancerous cells to grow and expand in some cases.

While more tests must be done before the treatment can be tested in humans, the researchers’ breakthrough offers hope to babies with this devastating disorder and their families. In the UK, as many as six pregnancies per year might be affected by ARC syndrome. Furthermore, the findings may promote improved understanding of why some treatments may cause cancer.

Corrected microbial family tree offers statistically sound model for how earliest life forms evolved

In this era of Big Data, the prevailing wisdom is that more information leads to better answers. However, a new Canadian study shows that in the hunt for life’s ancient ancestors, more data can actually lead to less truth. Published in the Proceedings of the National Academy of Sciences, the research by UdeM associate professor of computer science Miklós Csűrös reveals that standard methods for reconstructing the genomes of ancient microbes are being overwhelmed by an explosion of information.

This paradox causes current models to “hallucinate” evolutionary events—specifically, an implausibly high number of horizontal gene transfers—that are actually just statistical ghosts, the study shows.

In it, Csűrös identifies a crisis point in evolutionary biology: As researchers try to reconcile thousands of gene sequences across the entire tree of life, the actual evolutionary signal begins to vanish, replaced by mathematical noise.

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