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Polygenic Risk Scores Found to Predict Dilated Cardiomyopathy in General Population

Research indicates that dilated cardiomyopathy can be caused by the cumulative influence of potentially thousands of genes, not just one single genetic variant. The scientists suggest the findings could improve clinical genetic testing and better identify individuals at risk.

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Repair Proteins Collaborate in “Hubs” to Repair DNA Damage

Researchers have mapped the activity of repair proteins in individual human cells. The study demonstrates how these proteins collaborate in so-called "hubs" to repair DNA damage.

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snoRNA Target Discoveries Could Lead to Potential Therapeutic Strategies

Researchers developed a technology that identified thousands of previously unknown targets of small nucleolar RNAs, guide RNA molecules that steer chemical modifications to cellular ribosomal RNA targets. Their findings could have potential therapeutic applications.

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Nanoparticles for mRNA Delivery Get an Edge via Chemically Evolved Lipids

Moving beyond the speed/accuracy tradeoffs of current lipid nanoparticle engineering methods, a team of Penn scientists created high-performing lipid nanoparticles by using lipids that had been optimized by iterative directed chemical evolution.

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StockWatch: Moderna Seeks to Reassure Investors on RFK Jr.

Moderna’s top executives have sought to reassure investors they will be able to carry out the comeback plans announced in September and continue its development of mRNA-based vaccines should Robert F. Kennedy Jr. oversee HHS.

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Treehill CEO Sees Positives for Biopharma in Trump’s Re-Election

Ali Pashazadeh, the founder, chairman, and CEO of Treehill Partners, a London-based investment firm, discusses the likely effects of Donald Trump’s return to the White House in an exclusive interview with GEN Edge.

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Is Biopharma Ready for AI? Exploring Tech Strategies Across Large and Small Biopharma

In this GEN webinar, experts from AbbVie and Benchling will share insights into the current tech landscape, AI readiness, and the strategies for digital transformation shaping large and small biopharma.

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Novel Immunotherapy Attacks Glioblastoma from Multiple Angles

Researchers at the University of Basel and the University Hospital Basel have developed an immunotherapy in mice that not only attacks glioblastoma but also turns its microenvironment against it.

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Gene Therapy for Muscular Dystrophy Could Use StitchR to Assemble XL Gene from S Segments

New research from University of Rochester and elsewhere describes StitchR, an RNA-based technique for delivering large genetic payloads. Using this process, genes are delivered in two halves. They both generate mRNAs that are joined together to restore missing or inactive protein expression.

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Machine Learning Reveals Impact of Microbial Load on Gut Health and Disease

A new machine-learning model allows researchers to estimate microbial load based solely on microbial composition data, bypassing challenges to time-consuming and costly experimental methods for measuring microbial loads directly.

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Improving the Manufacture of mRNA Biologics

The COVID-19 pandemic engendered a rush to create massive numbers of doses of mRNA vaccine. To purify mRNA, industry often relies on standard methods designed for relatively small biologics such as monoclonal antibodies.

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Red Blood Cell Precursors Reverse Glutamine Metabolism to Remove Toxins, Avoid Disease

A previously unrecognized role for glutamine has been found in erythropoiesis. Early in differentiation, glutamine is broken down, but later it is synthesized to help RBCs cope with ammonium, a toxic byproduct of heme production.

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Quotient Sciences Installs GMP Biofoundry-in-a-Box for RNA Production

Centillion’s BiaB technology offers continuous flow-based manufacture and encapsulation of RNA, more flexibility with higher productivities at smaller footprints, and enables larger quantities of RNA drug substance to be split and encapsulated in portions.

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Memories in Stressed Mice Show Generalized Aversion, Possible PTSD Mechanism

Scientists uncovered that stress changes how our brain encodes and retrieves aversive memories, and provided potential mechanistic insights into stress-induced aversive memory generalization. Their preclinical results may point to treatment approaches for restoring appropriate memory specificity in PTSD.

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StockWatch: Vaccine, ETF Shares Drop as Trump Chooses RFK Jr. for HHS

Shares of several COVID-19 vaccine developers sank fast enough to reflect investor jitters about vaccine policy given RFK’s severe criticism of the jabs—whose development Trump’s first administration shepherded through “Operation Warp Speed.”

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