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Researchers detail groundbreaking Angelman syndrome development

Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome to advance into clinical development.

In a new article, published today in Science Translational Medicine, Dr. Scott Dindot, an associate professor and EDGES Fellow in the Texas A&M School of Veterinary Medicine and Biomedical Sciences’ (VMBS) Department of Veterinary Pathobiology, and his team share the process through which they developed this novel therapeutic candidate, also known as 4.4.PS.L, or GTX-102. Dindot is also the executive director of molecular genetics at Ultragenyx, which is leading the development of GTX-102.

Angelman syndrome (AS) is a devastating, rare neurogenetic disorder that affects approximately 1 in 15,000 per year; the disorder is triggered by a loss of function of the maternal UBE3A gene in the brain, causing , absent speech, movement or balance disorder, and seizures.

Gut microbiome can play key role in response to CAR-T cell cancer immunotherapy

Scientists from German Cancer Research Center (DKFZ), together with colleagues from Germany, Israel, and the U.S., have found that the gut microbiome may modulate the efficacy of CAR-T cellular immunotherapy CAR-T cells in patients with B cell lymphomas. Individualized microbiome information retrieved from patients’ gut microbiomes prior to initiation of CAR T therapy could accurately predict their subsequent responsiveness to therapy, but only in the condition that these patients were not pre-treated with broad spectrum antibiotics.

Increasing evidence from and preclinical experiments suggests that the gut microbiome may modulate the efficacy of T cell-driven cancer immunotherapies, such as immune checkpoint blockade. Immunotherapy with CD19 (CAR)-T cell has opened up new treatment options for with certain forms of refractory and relapsing B-cell leukemias or lymphomas. But the therapy is hampered by considerable heterogeneity in responses. Complete and long-term remission is only achieved in up to 40% of patients.

Researchers from multiple centers in Germany and the United States, led by Eran Elinav, director of the DKFZ-Weizmann Institute of Science Microbiome & Cancer Bridging division, have found that the gut microbiome may modulate the efficacy of CD19 CAR-T cell immunotherapy in patients with B cell leukemias and lymphomas.

Telomere shortening—a sign of cellular aging—linked to signs of Alzheimer’s in brain scans

Changes in the brain caused by Alzheimer’s disease are associated with shortening of the telomeres—the protective caps on the ends of chromosomes that shorten as cells age—according to a new study led by Anya Topiwala of Oxford Population Health, part of the University of Oxford, UK, published March 22 in the open-access journal PLOS ONE.

Telomeres on chromosomes protect DNA from degrading, but every time a cell divides, the telomeres lose some of their length. Short telomeres are a sign of stress and cellular aging, and are also associated with a higher risk of neurological and psychiatric disorders. Currently, little is known about the links between and changes that occur in the brains of people with neurological conditions. Understanding those relationships could offer insights into the biological mechanisms that cause neurodegenerative disorders.

In the new study, researchers compared telomere length in to results from brain MRIs and from more than 31,000 participants in the UK Biobank, a large-scale biomedical database and research resource containing anonymized genetic, lifestyle and from half a million UK participants.

Researchers identify neutrophils as major culprits in treatment resistance of pancreatic cancer

Researchers have shown for the first time exactly how immature neutrophils—white blood cells that are an important part of the immune system—are hijacked by pancreatic cancers to drive immunosuppression and treatment resistance. The study, led by investigators at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, is published in Cancer Discovery, a journal of the American Association for Cancer Research.

The paper describes a previously unrecognized signaling circuit in pancreatic cancer that instigates immunosuppression and tumor-promoting inflammation in the pancreatic tumor microenvironment, ultimately creating treatment resistance. The central regulator of this treatment resistance is neutrophil-derived TNF signaling. TNF, or , is a substance in the body that causes inflammation.

This is the first study to implicate immunosuppressive signaling from immature neutrophils—the earliest sentinels in developing pancreatic cancer—in this process, said Jashodeep Datta, M.D., associate director of Translational Research at the Sylvester Pancreatic Cancer Research Institute. Datta, whose laboratory at Sylvester led the research, is senior author of the multicenter study.

Unprecedented Breakthrough in Manipulating “Quantum Light”

For the first time, scientists at the University of Sydney.

The University of Sydney is a public research university located in Sydney, New South Wales, Australia. Founded in 1,850, it is the oldest university in Australia and is consistently ranked among the top universities in the world. The University of Sydney has a strong focus on research and offers a wide range of undergraduate and postgraduate programs across a variety of disciplines, including arts, business, engineering, law, medicine, and science.

Gravitas: Alarming rise of drug-resistant fungus | Candida Auris spreads in US

And just in time for the “Last of Us” series. 😂


A drug-resistant fungus Candida Auris is spreading in US hospitals at an alarming rate. Those with fragile immune systems are at risk. What happens if it enters your country or your neighbourhood? Should you be scared? Molly Gambhir reports.

#Gravitas #CandidaAuris #Fungus.

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Artificial neurons may repair damaged cells and circuits

Electronic neurons made from silicon mimic brain cells and could be used to treat autism1.

Researchers plan to use the technology in conjunction with machine learning to retrain damaged or atypical neurons and restore function in the brains of people with Alzheimer’s disease, autism or other conditions.

Another team attempted to make artificial neurons in 2015 from a conductive organic chemical, but that version oversimplified brain signaling and was too large to implant in a human brain2.