Inside Precision Medicine
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Exclusive news, features, and analysis on the research, tools, regulations, and applications driving precision medicine. From the publisher of @GENBio
Joined April 2014
A New Generation of #CRISPR Technology. At the age of 30, @janiceschen has not only completed a PhD with Nobel prize winner Jennifer Doudna, but she has also helped co-found her own successful #biotech company @mammothbiosci. Read more about her journey:
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Eric Topol, MD, in a keynote interview described his vision of how AI can help redefine cancer screening and diagnosis. Learn more:
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A drug for #idiopathicpulmonaryfibrosis designed by #AI specialist @InSilicoMeds has passed its safety goals in a phase I trial. Learn more:
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Natural Suppressor Mutations May Help Protect Us from Genetic Disorders. Learn more:
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The FDA has approved the first blood test that can help identify hundreds of potentially cancer-associated hereditary variants. Learn more:
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Researchers have identified a previously unknown signaling pathway in cells that protects their DNA while it is being copied—a discovery that suggests this pathway could be targeted for treating cancer. Read more:
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Researchers at the Cleveland Clinic Genome Center have identified potential genetic factors that could play a role in Parkinson’s disease, and have identified FDA-approved drugs that could be repurposed as new treatments.
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The Human Immunome Project Plans to Unlock the Immune System’s Secrets. The Human Immunome Project has revealed plans to decode and model the immune system by generating the largest immunological dataset to date. Learn more:
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#Alzheimers DNA Methylation Study Uncovers 84 New Genes Associated with the Disease. This meta-analysis, believed to be the largest of its type, reviewed six earlier DNA methylation studies of AD. Learn more:
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High quality #microscopy has captured mammalian translation of #SARSCoV2 viral RNA in unprecedented detail. The virus uses a ‘pseudoknot’ structure to create tension in the ribosome and promote frameshifting to translate viral RNA. Learn more:
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Blood Proteins Predict 10-Year Risk of 67 Diseases. Prediction models developed from blood proteins may be superior to clinical information for assessing the 10-year risk of 67 diseases, according to a study using data from the UK Biobank. Read more:
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Parkinson’s Test Predicts Disease Years before Symptoms. An eight-biomarker protein blood test can predict the onset of Parkinson's disease up to seven years in advance of motor symptoms. Learn more:
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Changes in Gut Microbiome Linked to Parkinson’s Disease Symptoms. People with Parkinson’s disease often develop gastric symptoms that can sometimes start years before they develop any movement problems. Learn more about this new study:
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Lipid-Lowering Drug Repurposed for #Parkinsons. #AI analysis of research papers has enabled the identification of a potential new role for the lipid-lowering drug probucol in Parkinson’s disease. Learn more:
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.@MayoClinic researchers are developing ways to employ #artificialintelligence to increase the detection of potentially cancerous polyps during colonoscopy. Read more:
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Scientists have linked a protein complex, GEMIN5, that controls RNA metabolism in neurons to a rare disease for the first time. Learn more:
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U.S. researchers have developed an #antiviral candidate drug targeting iron-sulfur clusters needed for #SARSCoV2 RNA replicase to function properly and for the virus to reproduce successfully. Read more: #COVID19
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Researchers developed a novel technique called CReATiNG (Cloning, Reprogramming and Assembling Tiled Natural Genomic DNA), offering a more straightforward and more cost-effective approach to constructing synthetic chromosomes. Learn more:
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A stem cell-based treatment for progressive multiple sclerosis (MS) achieved good results in a Phase I trial led by researchers at the University of Cambridge and the University of Milan Bicocca. Learn more:
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CRISPR-Based Epigenome Editing Platform Identifies Genes That Improve T-Cell Therapies for #Cancer Treatment. Scientists engineered a #CRISPR based #epigenomeediting platform and discovered a master regulator of the genome that reprograms T cells:
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It is estimated that rare diseases affect more than 30 million people in the US alone, and between 300 million and 400 million people. Here, we profile three such parents whose commitments to their cause are impacting the #raredisease community. Read more:
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Scientists Restore Brain's Waste Disposal System to Combat Aging. Reversing aging-related decline in the brain’s waste-clearing process, offers a potential new treatment for neurodegenerative diseases. Learn more:
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Digital Biomarker Company Imagene AI Secures $21.5M Investment. Imagene’s uses digital pathology images and proprietary algorithms to identify and classify patterns not discernible by human examination. Learn more:
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A new class of antibiotics has been discovered that is effective against multidrug-resistant bacteria, offering new opportunities to tackle these dangerous microbes. Read more:
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#GeneEditing Shows Potential for #CysticFibrosis. A single injection that deploys gene editing technology can correct a common mutation that causes cystic fibrosis and improve lung and gastrointestinal symptoms, research in mice has revealed. Learn more:
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Quicker and Cheaper Single Cell RNA Sequencing Method Developed. Austrian scientists have developed a method to individually barcode different cells before they are loaded onto a microfluidic chip. Learn more: #sequencing #rnasequencing
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Alzheimer’s Vaccine Shows Good Safety, Immunogenicity at Phase II. An experimental, therapeutic vaccine developed by Araclon Biotech showed good safety and immunogenicity results at Phase II. Learn more:
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Experts suggest that when a virus enters an immunosuppressed system, such as a cancer patient’s immune system, it lingers in the absence of adequate defenses and accumulates many mutations. Read more: #COVID19 #coronavirus
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Researchers have developed a new CRISPR-based technology that increases the recognition of cancer cells to the immune system. Learn more:
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A second patient with HIV has been cured with a stem cell transplant at Berlin’s Charité, which, in 2008, famously treated the “Berlin Patient,' who became the first person in the world to be cured of HIV. Learn more:
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Antioxidant Could Help Combat Drug Resistance in Breast Cancer. The antioxidant N-acetylcysteine could help overcome resistance to alpelisib in PI3Kalpha mutated #breastcancer patients. Learn more:
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High quality #microscopy has captured mammalian translation of #SARSCoV2 viral RNA in unprecedented detail. The virus uses a ‘pseudoknot’ structure to create tension in the ribosome and promote frameshifting to translate viral RNA. Learn more:
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Scientists uncover new biomarker predicting the clinical response to #CARTcelltherapy for #lymphoma treatment. Learn more:
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Fatty Acid Found in Palm Oil Can Promote #Cancer Metastasis. In a mouse model of human mouth and skin cancer, researchers found that dietary consumption of palmitic acid, but not oleic acid or linoleic acid, seemed to promote metastatic tumor growth:
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Blood Biomarker Flags Alzheimer’s Neurodegeneration. A new blood-based biomarker, brain-derived tau, can identify neurodegeneration specific to Alzheimer’s disease without requiring brain scans or samples of cerebrospinal fluid. Learn more:
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Mark your calendars for Friday, October 23rd and register today for our upcoming webinar--Optical Mapping in Rare Genetic Disease Diagnosis. Sponsored by .@bionanogenomics. Registration is free: #geneticdisease #genetics #opticalmapping
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PTC Nets AADC Gene Therapy Approval: First Brain-Delivered Gene Therapy in U.S. PTC Therapeutics’ gene therapy for AADC deficiency, marketed under the name Kebilidi, has received FDA approval.
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Single-cell RNA Sequencing Reveals Kidney Cancer Cellular Origins. Learn how: #sequencing #singlecellsequencing
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Bayer and NextRNA Therapeutics will jointly develop two first-in-class small molecule programs targeting long non-coding RNA. The deal is worth $500M+. Read more:
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Brazilian Researchers Develop Compound That Combats Multidrug-Resistant Bacteria in One Hour. A team of researchers has discovered a peptide that has shown promising action against six antibiotic-resistant bacterial pathogens. Learn more:
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Novel CRISPR Technique Holds Promise for Bespoke Cancer Treatment. A new type of CRISPR gene-editing technology has revealed the crucial cellular functions of noncoding RNAs and suggests they could be useful targets for personalized cancer treatments.
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Getting CRISPR to the Clinic. Although this gene-editing technology promises impressive improvements in healthcare, various obstacles must be addressed before large numbers of patients can benefit.
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Small Handheld Device Can Detect Bacterial Infections in Minutes. A USB stick-sized device can quickly and accurately detect bacterial urinary tract infections and has the potential to analyze blood or saliva and to detect viruses as well. Read more:
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RNA Sequencing Can Increase Diagnostic Rate for Rare Diseases. #RNAsequencing can help diagnose #rarediseases caused by changes in #geneexpression that are not detectable using standard #genomicsequencing methods. Learn more:
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Junk DNA Sequence a Likely Driver of Telomerase Gene Activity. Researchers found that deleting VNTR2-1 from #cancer cells—both in a human cell line and in mice—caused telomeres to shorten, cells to age, and tumors to stop growing. Learn more:
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Researchers based in Madrid have identified a microRNA biomarker that can accurately diagnose patients with the cardiac inflammatory condition #myocarditis, something that has been hard to diagnose accurately in the past. Learn more:
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Biomarkers Found That May Predict Risk of Common Pregnancy Complications. Microbiome-related biomarkers that could predict three common and dangerous pregnancy complications have been identified . Read more:
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Doubling of Y Chromosome Doubles Autism Risk . Risk of autism appears to be linked to the Y chromosome, a Geisinger study found, which may explain the greater prevalence of Autism spectrum disorder (ASD) in males.
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Researchers at the Icahn School of Medicine at Mount Sinai say they have developed a novel platform to overcome one of the biggest challenges in brain disease treatment: getting macromolecule therapeutics to cross the blood-brain barrier (BBB).
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Lab-on-a-Chip Genetic Test Can Identify a Virus within Three Minutes. A lab-on-a-chip genetic diagnostic test can provide lab-quality results to identify a virus within three minutes. Learn more:
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ICL and Oxford Nanopore Technologies have developed a DNA barcoding method that allows nanopore sequencing to identify dozens of biomarkers in one test. The method shows promise to identify specific biomarkers of diseases such as cancer. Learn more:
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The FDA has approved the first blood test that can help identify hundreds of potentially cancer-associated hereditary variants. Learn more:
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Researchers at @UCSF have discovered how #cancer cells evade targeted therapy using an escape mechanism called phenotype switching, potentially explaining the failure of precision therapies for #glioblastoma to date. Read more:
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A new study confirms previous suspicions that #Parkinsonsdisease may have an inflammatory or autoimmune component, as those with the condition have a notably different gene expression profile in their T cells compared with healthy controls. Learn more:
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Five RNA Therapeutics Startups to Watch in 2022. Five RNA therapeutics startups to watch that have opened their doors since 2020 and are developing the next generation of RNA medicines. Learn more:
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Mitochondrial DNA Triggers Inflammation in #SickleCellAnemia Patients. The red blood cells of people with this chronic condition seem to accumulate mitochondria, which are absent from healthy blood cells. Learn more:.
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Innovative research from @uottawa suggests that edaravone, a drug currently used to treat ALS, could be repurposed to fight glioblastoma. The study demonstrates that edaravone targets brain tumor stem cells, responsible for tumor growth and recurrence.
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THIS FRIDAY, join us for our upcoming free webinar--Optical Mapping in Rare Genetic Disease Diagnosis. Sponsored by .@bionanogenomics. Register today: #geneticdisease #genetics #opticalmapping #webinar #raredisease
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Targeting a New Receptor on T Cells to Improve Immunotherapy. A new anticancer immunotherapy targets the activin receptor 1C on T cells to boost immune system attack against cancer cells. Read more:
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A single mutation in the H5N1 “bird flu” virus could enhance its ability to attach to human cells, raising concerns about the potential for human-to-human transmission, according to scientists at Scripps Research.
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Researchers discovered eliminating the gene SRC-3 in breast and prostate #cancer animal models led to a lifelong anti-cancer response. Also, transferring immune cells without SRC-3 to animals with breast cancer tumors killed the tumor. Learn more:
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.@RevealGenomics’ ctDNA- and #machinelearning based liquid biopsy predicts response to certain therapies and survival in #breastcancer study. Read more:
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New Study Illuminates Bacteria’s Role in #PancreaticCancer. Live bacteria from cystic pancreatic lesions, which are precursors to pancreatic cancer, have been found to have an over-representation of Gammaproteobacteria and Bacilli. Learn more:
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Scientists find a new way to track peripheral artery disease (PAD). Learn more:
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Sensitive Tumor DNA Test Effective at Predicting Lung Cancer Progression. A test developed by U.S.-biotech Personalis can pick up small amounts of lung cancer tumor DNA in the blood and predict patient outcomes.
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Scientists at @UTokyo_News_en have designed an artificial hairpin-like DNA molecule which is able to target and kill #cancer cells by binding to microRNA (miRNA) molecules that are overproduced in certain cancers. Learn more:
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Dana-Farber Scientists Develop PET Probe to Image Inflammation with High Sensitivity. The approach uses a probe targeting CD45, a protein marker present on all immune cells, to provide clear image of immune system organs.
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AI Tool Can Predict Hospitalized Patients at Risk of Deterioration. An AI-driven early warning tool can help predict which hospitalized patients are about to deteriorate and allow them to get fast treatment. Learn more:
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.@IncellDx Receives CE Mark for First #LongCOVID Diagnostic. The test, which demonstrated greater than 90% accuracy across #COVID strains is set to launch in the European Union this month. Learn more: .
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New Technique Improves T Cell-Based Immunotherapies for Solid Tumors. A new technique that removes a sugar on the outside of solid tumor cancer cells enhances the ability of T cells to recognize and kill tumor cells. Learn more:
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#DeepLearning Model Can Predict Adverse Drug-Drug Interactions. A deep learning model based on gene expression data can accurately predict whether a drug will adversely interact with other drugs. Learn more:
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Changes in Gut Microbiome Linked to Parkinson’s Disease Symptoms. People with Parkinson’s disease often develop gastric symptoms that can sometimes start years before they develop any movement problems. Learn more about this new study:
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A new technology for delivering nucleic acid therapeutics—such as siRNA, ASOs, and mRNA—to bone marrow cells appears to overcome long-posed challenges of stability and precision targeting. @radboudumc @TUeindhoven.
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Semaglutide may significantly lower the risk of Alzheimer’s disease in people with type 2 diabetes, compared to other diabetes drugs.
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The digital edition of the December issue is now available. We find ourselves in this issue focusing on the burgeoning field of #proteogenomics and the advancement of single-cell technologies being used to delve into the proteome more deeply. Read now:
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First Gene-Modified Pig Liver Xenotransplantation Into Human Recipient. This successfully transplant, without signs of rejection, marks a significant milestone in addressing the critical shortage of liver organ donors. #XijingHospital .
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Researchers at @umichmedicine report the development of new biomarkers that help in the early screening of patients for the development of #myocarditis following ICI #cancer therapy. Learn more:
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Gene Therapy Targeting Myddosome May Prevent Retinal Degeneration. Large intracellular signaling complexes called Myddosomes are implicated in the progression of age-related macular degeneration (AMD). Learn more:
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A research team at the Albert Einstein College of Medicine has made a significant discovery that could enhance the effectiveness of stem-cell transplants, a critical treatment for patients with cancer, blood disorders, or autoimmune diseases. Learn more:
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Multi-Omics Study Reveals Four Types of Alzheimer’s. A cross-omics approach has identified four distinct molecular profiles of Alzheimer’s Disease, one of which is particularly serious. Learn more:
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A biomarker has been found that distinguishes whether ductal carcinoma in situ, a sometimes benign #breastcancer, will become invasive. Learn more:
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Scientists at @UTokyo_News_en have designed an artificial hairpin-like DNA molecule which is able to target and kill #cancer cells by binding to microRNA (miRNA) molecules that are overproduced in certain cancers. Learn more:
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Researchers at the Johns Hopkins University School of Medicine and the Johns Hopkins Kimmel Cancer Center have identified a protein called METRNL that depletes energy from T cells, severely limiting their ability to fight cancer. Read more: .
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Designer Peptide Halts #Neurodegeneration. Researchers have designed a peptide that can block the toxic effects of a protein linked with neurodegenerative disease, which could potentially be delivered via nasal spray. Learn more:
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Researchers at @ColumbiaMed have engineered probiotic bacteria to act as personalized cancer vaccines that can activate the immune system to target and destroy cancer cells. The new approach harnesses the natural tumor-targeting properties of bacteria.
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Vitamin D May Improve Cancer Immunity by Altering the Gut Microbiome. Researchers have discovered that vitamin D can increase the amount of a certain type of gut bacteria, providing better immunity to cancer. Learn more:
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Since the start of the pandemic, mRNA vaccine development to combat #COVID19 has happened extremely quickly, but can these scientific advances can have a positive impact on the development of cancer vaccines? Read more:
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In Another Hemophilia Advance, @pfizer Reports Positive Results for #GeneTherapy. Learn more:
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AI-Designed Protein Used to “Wake Up” Silenced Genes. By combining #CRISPR technology with a protein designed using #artificialintelligence, individual dormant genes were awaked by disabling the chemical “off switches” that silence them. Learn more:
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Scientists have discovered a new #genetic disease, which causes some children’s brains to develop abnormally, resulting in delayed intellectual development and often early onset cataracts. Affected children also developed microcephaly. Learn more:
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AI-Powered Liquid Biopsy Delivers Ultrasensitive Cancer Detection. An AI-powered test shows unprecedented sensitivity in predicting cancer recurrence from blood samples. Learn more:
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Researchers ID Four Distinct Subtypes of Autism. A brain study has found that people with #autism can be classified into four distinct subtypes based on their brain activity and behavior. Learn more:
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A new method by which cells turn genes on and off has been described by @UTSWNews researchers in Cell. Their findings could lead to new ways of controlling gene regulation and possibly new treatments for various diseases. Learn more:
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Could a Tiny Molecule Be the New Target for Alzheimer's Disease?. Scientists have discovered that a tiny molecule called microRNA-132 could pose as a potential therapeutic target for #Alzheimersdisease. Learn more:
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ALS and Frontotemporal Dementia Linked to Mutation Causing Inflammation. A mutated gene is linked to Amyotrophic lateral sclerosis and frontotemporal dementia through the overproduction of Interleukin-17A. Learn more:
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Organoid Blood-Brain Barrier Could Improve Neurological Treatments. A cellular model of the blood-brain barrier could help develop better drugs and diagnostics for conditions such as Alzheimer's disease. @SeoulNatlUni @postech2020.
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AstraZeneca is the latest big pharma to partner with a company using artificial intelligence (AI) to help design drugs in collaboration with U.S.-based Absci to design a cancer-fighting antibody drug. Learn more:
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Blood Test Identifies Early Brain Changes Linked to Dementia Risk. Researchers uncover PIGF as a novel blood-based biomarker for detecting early brain changes leading to cognitive decline and dementia.
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