Tag: ALS

King’s College London hiring Research Associate in Translational Bioinformatics in London, England, United Kingdom

Job DescriptionThis is a Pre-Fellowship for early career researchers wanting to establish a career in MND translational research and become MND research leaders of the future. The postholder will have a leading role in an exciting new project funded by Motor Neurone Disease Association and MND Scotland. The project is…

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Unravelling cell type-specific responses to Parkinson’s Disease at single cell resolution | Molecular Neurodegeneration

Single nucleus RNA-seq reveals cell type heterogeneity in human SNpc We sampled SNpc from post-mortem human brains of 15 sporadic Parkinson’s disease (PD) patients and 14 Control individuals (see Supplementary Table 1 for full pathology reports). Using a 10X Genomics Chromium platform, we performed single nucleus RNA-seq (snRNA-seq) on more than…

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ALS Agent PrimeC Moves to Phase 3, FDA Clears IND for AB126, APOE Carrier Status Impacts ARIA

WATCH TIME: 3 minutes Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Shortly after positive topline data from the phase 2 PARADIGM study of PrimeC was released, NeuroSense Therapeutics has announced that the FDA has confirmed its development plans for a phase 3 pivotal trial with…

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Altered quorum sensing and physiology of Staphylococcus aureus during spaceflight detected by multi-omics data analysis

CDC. (ed CDC) (U.S. Department of Health and Human Services, CDC; 2019, Atlanta, GA, 2019). Krismer, B., Weidenmaier, C., Zipperer, A. & Peschel, A. The commensal lifestyle of Staphylococcus aureus and its interactions with the nasal microbiota. Nat. Rev. Microbiol. 15, 675–687 (2017). Article  CAS  PubMed  Google Scholar  Somerville, G….

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Food Authenticity Market worth $12.23 billion by 2030, growing

Food Authenticity Market | 360iResearch The “Food Authenticity Market by Type (DNA Sequencing/Barcoding, Immunoassay-Based/Enzyme-Linked Immunosorbent Assay (ELISA), Isotope Methods), Category (Adulteration Tests, Allergen Testing, False Labelling), Application – Global Forecast 2023-2030” report has been added to 360iResearch.com‘s offering. The Global Food Authenticity Market to grow from USD 7.27 billion in…

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Elevated peripheral levels of receptor-interacting protein kinase 1 (RIPK1) and IL-8 as biomarkers of human amyotrophic lateral sclerosis

Ethics statement All the animal experiments were performed in accordance with the Guide for the Care and Use of Laboratory Animals and were approved by the Animal Care Committee of Three Gorges University, China (2019060B). The approval of primidone off-label prescription for ALS patients was obtained from the Ethics Committee…

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The Protective Mechanism of TFAM on Mitochondrial DNA and its Role in Neurodegenerative Diseases

Annesley SJ, Fisher PR (2019) Mitochondria in health and disease. Cells 8(7). doi.org/10.3390/cells8070680 Cannino G, Ferruggia E, Luparello C, Rinaldi AM (2009) Cadmium and mitochondria. Mitochondrion 9(6):377–384. doi.org/10.1016/j.mito.2009.08.009 Article  CAS  PubMed  Google Scholar  Bonora M, Missiroli S, Perrone M, Fiorica F, Pinton P, Giorgi C (2021) Mitochondrial control of genomic…

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Transition of allele-specific DNA hydroxymethylation at regulatory loci is associated with phenotypic variation in monozygotic twins discordant for psychiatric disorders | BMC Medicine

Lichtenstein P, Yip BH, Bjork C, Pawitan Y, Cannon TD, Sullivan PF, Hultman CM. Common genetic determinants of schizophrenia and bipolar disorder in Swedish families: a population-based study. Lancet. 2009;373(9659):234–9. Article  PubMed  Google Scholar  Maurano MT, Humbert R, Rynes E, Thurman RE, Haugen E, Wang H, Reynolds AP, Sandstrom R,…

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The biggest private biotech investments in November 2023

The companies Cambrian Innovation, Terremoto Biosciences, and VectorY Therapeutics bagged the biggest biotech investments in November 2023. Around the world, oncology and central nervous system players attracted the biggest funding rounds. As we edge closer to Christmas, biotech fundraising appears to be drying up a bit; there was a significant…

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Cathleen Lutz, MD, on the Potential of Stathmin-2 as an ALS-Specific Biomarker

Amyotrophic lateral sclerosis (ALS), a neuromuscular disease characterized by selective degeneration of upper and lower motor neurons, is currently lacking validating disease-specific biomarkers. The field has seen significant progress in expanding the therapeutic options available to patients, highlighted by the approvals of AMX0035 (Relyvrio; Amylyx) and tofersen (Qalsody; Biogen), the…

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Alzheimer’s blood test could hit the market in early 2024, researchers say

Could a simple blood test detect Alzheimer’s disease years before symptoms appear? New research from Resonant, a Utah biotech company that develops diagnostic tests for neurodegenerative diseases, suggests it may be possible. Researchers said its new test achieved 100% accuracy in identifying patients with Alzheimer’s disease and individuals with mild cognitive impairment (MCI) who went…

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Remote Bioinformatics Analyst II – Remote | WFH in United States

We are a dynamic and collaborative international bioinformatics team dedicated to advancing Neuroscience research. Our focus lies in harmonizing internal OMICS data sets, ensuring effective data storage according to FAIR data principles (Findable, Accessible, Interoperable, and Reusable), and broadening our knowledge base for future integrative analyses. ** Project Objective And…

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Mitophagy in human health, ageing and disease

Palikaras, K., Lionaki, E. & Tavernarakis, N. Mechanisms of mitophagy in cellular homeostasis, physiology and pathology. Nat. Cell Biol. 20, 1013–1022 (2018). Article  CAS  PubMed  Google Scholar  Palikaras, K., Lionaki, E. & Tavernarakis, N. Coordination of mitophagy and mitochondrial biogenesis during ageing in C. elegans. Nature 521, 525–528 (2015). Article …

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Potential use of iPSCs for disease modeling, drug screening, and cell-based therapy for Alzheimer’s disease | Cellular & Molecular Biology Letters

Tarawneh R, Holtzman DM. The clinical problem of symptomatic Alzheimer disease and mild cognitive impairment. Cold Spring Harb Perspect Med. 2012;2(5): a006148. Article  PubMed  PubMed Central  Google Scholar  van der Flier WM, Scheltens P. Epidemiology and risk factors of dementia. J Neurol Neurosurg Psychiatry. 2005;76(suppl 5):v2-7. Article  PubMed  PubMed Central …

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Refubium – Liquid Biopsy – Blood-based biomarkers for the molecular analysis of cancer

Tissue analysis is the current gold standard for cancer diagnosis and characterization, although it may neither fully represent spatial tumor heterogeneity nor clonal evolution under treatment pressure. The analysis of circulating tumor cells (CTCs) and cell-free DNA (cfDNA) holds great potential to partially overcome this limitation. One major uncertainty is,…

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The role of Centrosomal protein 83 (CEP83) in kidney progenitor differentiation from human induced pluripotent stem cells (hiPSCs)

Background: Centrosomal protein 83 (CEP83) is a component of the distal appendages proteins (DAPs) of centrioles, which is necessary for the assembly of primary cilia. Primary cilia play an essential role in early embryonic development, organ development, and tissue homeostasis. Loss of many ciliary proteins in mice models results in…

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Head of the Medical Bioinformatics platform (m/f/d) – Kiel

Das Universitätsklinikum Schleswig-Holstein (UKSH) verbindet internationale Spitzenforschung mit interdisziplinärer Krankenversorgung. Wir sind einziger Maximalversorger und größter Arbeitgeber des Landes. Unsere mehr als 14.500 Mitarbeiter*innen stellen eine höchst individuelle Versorgung sicher – unverzichtbar für die Menschen in Schleswig-Holstein.The Institute of Clinical Molecular Biology (IKMB)The Medical Faculty together with the Institute of…

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The Rauvolfia tetraphylla genome suggests multiple distinct biosynthetic routes for yohimbane monoterpene indole alkaloids

Genome sequencing and assembly of R. tetraphylla After DNA extraction from young leaves and sequencing, the R. tetraphylla genome was first assembled into 1008 contigs with an N50 of 3.7 Mb. After haplotigs removal and a final pilon polishing, the 364,945,498 bp final assembly was distributed across 76 scaffolds with an N50…

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A Deep Dive into Trends, Players, and Growth

The gene therapy market has evolved into a juggernaut in the healthcare sector, displaying an extraordinary trajectory of growth. As of 2023, the gene therapy market has surged to a remarkable value of USD 6.36 billion, and the projection for the forthcoming years is nothing short of astonishing. Forecasts indicate…

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Sequencing 101: Tandem repeats – PacBio

In the field of genetics, the concept of tandem repeats has been both scientifically fascinating, experimentally challenging, and motivating for technology development. As DNA sequencing technologies and analysis tools have evolved, scientists are now able to reveal the secrets hidden within these repetitive sequences, shedding light on their significance in…

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Help 36 million people in Europe with rare diseases

image: @ Hydromet | iStock Baiba Miltoviča, President of the Section for Transport, Energy, Infrastructure and the Information Society at EESC, lifts the lid on rare diseases, charting a European mission to help 36 million people in the EU Europe needs a strategy to give people with rare diseases the…

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BrainStorm Cell Therapeutics (BCLI) Announces In-Person Meeting with the FDA to Discuss Confirmatory Phase 3 Trial for NurOwn in ALS

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that the US Food & Drug Administration (US FDA) has granted the company a meeting to discuss the regulatory path forward for NurOwn® in amyotrophic lateral sclerosis (ALS). The meeting is…

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Morphological evolution of bird wings follows a mechanical sensitivity gradient determined by the aerodynamics of flapping flight

Claramunt, S., Derryberry, E. P., Brumfield, R. T. & Remsen, J. V. Ecological Opportunity and Diversification in a Continental Radiation of Birds: Climbing Adaptations and Cladogenesis in the Furnariidae. Am. Naturalist 179, 649–666 (2012). Article  Google Scholar  Derryberry, E. P. et al. Lineage diversification and morphological evolution in a large-scale…

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New therapeutic strategy to reduce neuronal death in amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects neurons in the brain and spinal cord causing loss of muscle control. A study by the University of Barcelona has designed a potential therapeutic strategy to tackle this pathology that has no treatment to date. It is a molecular trap…

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BrainStorm Cell Therapeutics to Present at 6th Annual ALS Research Symposia

Presentation on NurOwn Phase 3 data demonstrating evidence of biological effect in ALS; Biomarker data reinforce clinical outcomes NEW YORK, Nov. 17, 2023 /PRNewswire/ — BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced a podium presentation and panel discussion at the…

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Novel Therapy Strategy Limits Neuron Death in ALS

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects neurons in the brain and spinal cord causing loss of muscle control. A study by the University of Barcelona has designed a potential therapeutic strategy to tackle this pathology that has no treatment to date. It is a molecular trap…

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FDA OKs Neurosense’s CMC for PrimeC ahead of Phase 3 trial

The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study. The Phase 3 trial of PrimeC is due to start in 2024 pending…

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Neurosense Therapeutics (NRSN) Receives U.S. FDA Confirmation of CMC Strategy for PrimeC in Preparation for Pivotal Phase 3 in ALS and Commercial Readiness

NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) (“NeuroSense”), a company developing treatments for severe neurodegenerative diseases, today announced it has concluded a successful Type D meeting with the U.S. Food and Drug Administration (FDA) for PrimeC in the treatment of amyotrophic lateral sclerosis (ALS). FDA Type D meetings are focused on a…

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New Biomarker Promises Earlier Diagnosis of Alzheimer’s Disease

Credit: Naeblys / Getty Images Renew Biotechnologies’ subsidiary Resonant recently published research in the journal Frontiers in Neurology of a new blood biomarker that promises earlier diagnosis of Alzheimer’s disease (AD). The approach, which centers on the use of cell-free DNA (cfDNA) analyzes differential methylation regions (DMRs) to identify biomarkers…

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Uncertain futures and unsolicited findings in pediatric genomic sequencing: guidelines for return of results in cases of developmental delay | BMC Medical Ethics

Guidelines for informed consent & return of results To tailor informed consent and return of UFs to the situations of children undergoing WES in trio-analysis (in which the parents’ and child’s DNA is sequenced and interpreted) for clarifying a DD, the guidelines we propose contain two important features. Firstly, instead…

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[Molecular diagnostics for vitreoretinal lymphoma]

Review doi: 10.1007/s00292-023-01251-z. Online ahead of print. [Article in German] Irina Bonzheim  1 , Julia Salmerón-Villalobos  2 , Daniela Süsskind  3 , Peter Szurman  4 , Florian Gekeler  3   5 , Martin S Spitzer  6 , Itziar Salaverria  2 , Elias Campo  2 , Sarah E Coupland  7 , Leticia Quintanilla-Martinez  8 , Falko Fend  8 Affiliations Expand Affiliations…

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Studies Identify Novel Underpinnings of Genetic ALS

Evangelos Kiskinis, PhD, associate professor in the Ken and Ruth Davee Department of Neurology’s Division of Neuromuscular Disease and of Neuroscience, was senior author of studies published in Science Advances and Cell Reports. A pair of recent studies from the laboratory of Evangelos Kiskinis, PhD, associate professor in the Ken…

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Two sympatric lineages of Australian Cnestus solidus share Ambrosiella symbionts but not Wolbachia

Andersen HF, Jordal BH, Kambestad M, Kirkendall LR (2012) Improbable but true: the invasive inbreeding ambrosia beetle Xylosandrus morigerus has generalist genotypes. Ecol Evol 2:247–257 Article  PubMed  PubMed Central  Google Scholar  Arif S, Gerth M, Hone‐Millard WG, Nunes MDS, Dapporto L, Shreeve TG (2021) Evidence for multiple colonisations and Wolbachia…

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Resonant Publishes Groundbreaking Alzheimer’s Disease Research With the Potential to Revolutionize Neurodegenerative Diagnostics

PROVO, Utah, Nov. 10, 2023 /PRNewswire/ — Resonant, a subsidiary of Renew Biotechnologies aimed at releasing novel diagnostics for neurodegenerative diseases, recently published research revealing a novel blood biomarker of neurodegeneration. The technology achieved a remarkable 100% accuracy in identifying patients with Alzheimer’s disease and individuals with Mild Cognitive Impairment (MCI),…

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Your weekly fix of the latest biotech news: November 10, 2023

You are reading Labiotech’s weekly roundup of the latest biotech news. Korro Bio and Frequency Therapeutics close merger Last Friday, Korro Bio announced the completion of the previously announced business combination between Frequency Therapeutics, Inc. and the entity formerly known as Korro Bio. The combined company will now operate under…

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MaaT Pharma Provides Third Quarter 2023 Business Update and Reports Financial Results

MaaT Pharma Provides Third Quarter 2023 Business Update and Reports Financial Results Independent Data Safety Monitoring Board (DSMB) reviewing the Phase 3 ARES trial evaluating MaaT013 in aGvHD concluded on a positive benefit/risk ratio based on a good safety profile and positive preliminary efficacy results with an Overall Response Rate…

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Mechanism and therapeutic potential of targeting cGAS-STING signaling in neurological disorders | Molecular Neurodegeneration

Hemmi H, et al. A toll-like receptor recognizes bacterial DNA. Nature. 2000;408(6813):740–5. Article  CAS  PubMed  Google Scholar  Unterholzner L, et al. IFI16 is an innate immune sensor for intracellular DNA. Nat Immunol. 2010;11(11):997–1004. Article  CAS  PubMed  PubMed Central  Google Scholar  Chiu YH, Macmillan JB, Chen ZJ. RNA polymerase III detects…

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Developed the most comprehensive database for

image:  Main page of the data base which includes the complete proteomes of the twelve model organisms. view more  Credit: IBB-UAB Researchers at the IBB-UAB have developed the most comprehensive database available to date to help understand the basis of protein aggregation, a phenomenon associated with ageing and several pathologies….

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Get It Recruit – Healthcare hiring Bioinformatics Analyst II – Remote | WFH in Chicago, IL

Are you passionate about leveraging your data expertise to make a meaningful impact on neuroscience research? We are seeking a dedicated individual to join our international bioinformatics team in a remote role (EST time zone) as a Data Harmonization and Analysis Specialist. In this position, you will play a pivotal…

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Bioinformatics Data Analyst job in Chicago, IL at Get It Recruit

Summary Description: We are seeking a highly skilled and motivated individual to join our team on an exciting project focused on harmonizing and curating internal OMICS datasets. This project plays a vital role in facilitating future integrative analyses in the field of neuroscience and furthering our understanding of diseases like…

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When Glia Meet Induced Pluripotent Stem Cells (iPSCs)

Abstract The importance of glial cells, mainly astrocytes, oligodendrocytes, and microglia, in the central nervous system (CNS) has been increasingly appreciated. Recent advances have demonstrated the diversity of glial cells and their contribution to human CNS development, normal CNS functions, and disease progression. The uniqueness of human glial cells is…

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Does A315T mutation of TDP43 causes cytoplasmic aggregates in hek293 cells?

The A315T mutation of TDP43 does not cause cytoplasmic aggregates in HEK293 cells. The A315T mutation is one cause of familial amyotrophic lateral sclerosis (ALS) and is associated with cytoplasmic aggregates in neurons of ALS patients. However, the formation of cytoplasmic aggregates in HEK293 cells is not observed in the…

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papain family cysteine protease containing protein, maker-scaffold1702_size30647-snap-gene-0.14 (gene) Tigriopus kingsejongensis

Associated RNAi Experiments Homology BLAST of papain family cysteine protease containing protein vs. L. salmonis genes Match: EMLSAG00000006045 (supercontig:LSalAtl2s:LSalAtl2s327:400616:404607:-1 gene:EMLSAG00000006045 transcript:EMLSAT00000006045 description:”augustus_masked-LSalAtl2s327-processed-gene-4.0″) HSP 1 Score: 590.497 bits (1521), Expect = 0.000e+0Identity = 283/525 (53.90%), Postives = 368/525 (70.10%), Query Frame = 0 Query: 49 GHVARPLGKSPPNFVRDPPPRTTPPAQWLWNNVNETNFLTVSRNQHLPTYCGSCWAHAATSSLSDRIKIARQGAWPDINLAPQVLISCGPGDGCHGGEAGDANAYMHAQGITDETCSIYRARGQDNGLPCSKLEICSTCE—SKCYQPQHFFTYRVDEFHDVEGESNGEQEANMMAEIHHRGPISCGIAVTQALV-NYTGGLFHDKTGAQEIDHDISVVGYGVDEGTQEKYWLIRNSWGTYWGEQGFFRLIRGVNNLGIESGTCSWATPADTWSDAARE—RAAILSNEITLQKP——LWKQLWTVVADFVDNTRDTDLFRRLKLMQKGCKKLSSPRVPVVNIRPRPQDYVSTADLPEALDWRSVNGTNFLSWSVNQHLPVYCGSCWAQAGLSSLADRFTIADRKRFANLALSVQYILNCQAGGSCHGGDAFPLYAFIQKQGVPDVTCQPYEALDEGPLTDCSKPSKLVCKDCTWPPPEPGQEGNCWAKEKFHRYYVDEYNGVEGADNMKKEILERGPVT 560 GH+ R G+…

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The History of Brainstorm Cell Therapeutics Inc

Brainstorm Cell Therapeutics Inc. is a renowned biotechnology company that has made significant contributions to the field of stem cell research. With a rich history spanning over two decades, the company has emerged as a pioneer in developing innovative therapies for neurodegenerative diseases. Let’s delve into the fascinating journey of…

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Revolutionising neuromuscular disease research through 3D modelling Labmate Online

A recent case study published by AMSBIO highlights ground-breaking research in neuromuscular diseases. Led by Dr Roger Kamm at the Massachusetts Institute of Technology (MIT), this project focuses on developing a 3D microfluidic neuromuscular platform for modelling Motor Neuron Diseases (MNDs). MNDs, particularly amyotrophic lateral sclerosis (ALS), remain a significant…

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Neuromuscular disease research | Scientist Live

AMS Bio has published a case study that describes how a team of scientists, led by Dr. Roger Kamm from the Massachusetts Institute of Technology (MIT), has developed a protocol for the fabrication of a 3D microfluidic neuromuscular platform that can be used to model motor neurone diseases (MNDs). For one…

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Bioconductor – Bioconductor 3.18 Released

Home Bioconductor 3.18 Released October 25, 2023 Bioconductors: We are pleased to announce Bioconductor 3.18, consisting of 2266 software packages, 429 experiment data packages, 920 annotation packages, 30 workflows and 4 books. There are 69 new software packages, 10 new data experiment packages, 8 new annotation packages, no new workflows,…

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Bioinformatics Scientist (Data Science) – NeuroTechX

  Bioinformatics Scientist (Data Science) Research – Data Sciences           ·            Cambridge, MA             ·          Full time    About Voyager Therapeutics  Voyager Therapeutics (Nasdaq: VYGR) is a biotechnology company dedicated to breaking through barriers in…

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Gene editing approaches for the treatment of Huntington’s disease and ALS by Huidagene Therapeutics

Neurology/Psychiatric Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT…

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BrainStorm making cuts to support new trial of NurOwn for ALS

BrainStorm Cell Therapeutics is making strategic adjustments — including cuts in its workforce — to accelerate the clinical development of NurOwn as an amyotrophic lateral sclerosis (ALS) treatment. The decision follows the recent withdrawal of its regulatory application with the U.S. Food and Drug Administration (FDA) seeking the therapy’s approval,…

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BrainStorm Cuts 30% of Staff to Refocus on ALS Cell Therapy’s New Phase 3 Trial

BrainStorm Cell Therapeutics is mapping out the path forward for its experimental amyotrophic lateral sclerosis (ALS) therapy, and while those plans include a new Phase 3 clinical trial the biotech is slimming down its operations in order to pay for it. About 30% of BrainStorm’s staff will be laid off,…

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Velotix Data Security Platform Integrates with Google Cloud’s BigQuery for Comprehensive and Automatic Data Access Control

BigQuery Users benefit from Automatic and AI Powered Data Discovery, Access Management, and Policy Management NEW YORK, Oct. 24, 2023 /PRNewswire/ — Velotix, a leading data security company, announced today an integration with Google Cloud’s BigQuery, a serverless enterprise data warehouse, to provide AI-driven data protection and access management for…

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Genome-wide analyses identify NEAT1 as genetic modifier of age at onset of amyotrophic lateral sclerosis | Molecular Neurodegeneration

van Es MA, Hardiman O, Chio A, Al-Chalabi A, Pasterkamp RJ, Veldink JH, van den Berg LH. Amyotrophic Lateral Sclerosis. Lancet (London England). 2017;390:2084–98. Article  PubMed  Google Scholar  Liu MS, Cui LY, Fan DS. Age at onset of Amyotrophic Lateral Sclerosis in China. Acta Neurol Scand. 2014;129:163–7. Article  CAS  PubMed …

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TUDCA given ALS patients at higher dose seen to extend survival

People with amyotrophic lateral sclerosis (ALS) who are treated daily with 1,000 mg or higher doses of tauroursodeoxycholic acid (TUDCA) tend to live longer than those given standard care alone, according to an analysis of registry data. “In our ‘real-world’ study, patients who received TUDCA at the higher dose survived…

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Datenauswertung Statistik Beratung R Studio SPS…

Hey, Als Masterand der klinischen Psychologie und Psychotherapie mit durchweg sehr guten Leistungen, sowie herausragenden Leistungen in den empirischen Forschungsmethoden und Statistik, sowie langjähriger und diverser Berufserfahrung in der Forschung als auch Beratung in allen Angelegenheiten empirischer Forschungsarbeiten berate ich Dich gerne kostengünstig bei allen Themen rund um die empirische…

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Renowned ALS Researcher Confirms The Efficacy Of NeuroSense Therapeutics’ Lead Drug Candidate, PrimeC

HERZLIYA, ISRAEL / ACCESSWIRE / October 19, 2023 / Induced pluripotent stem cells (iPSCs) have emerged as a promising tool in the field of research and therapy for neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS). ALS is a progressive disorder that affects motor neurons, leading to muscle weakness, paralysis…

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Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium

Additional details regarding trial enrollment and eligibility criteria will be shared upon initiation of the trial, which is anticipated to start by the end of 2023. AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), which was granted full approval as…

Continue Reading Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium

Renowned ALS Researcher Confirms The Efficacy Of NeuroSense Therapeutics’ Lead Drug Candidate, PrimeC – NeuroSense Therapeutics (NASDAQ:NRSN)

Induced pluripotent stem cells (iPSCs) have emerged as a promising tool in the field of research and therapy for neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS). ALS is a progressive disorder that affects motor neurons, leading to muscle weakness, paralysis and ultimately death. iPSCs are adult cells that have…

Continue Reading Renowned ALS Researcher Confirms The Efficacy Of NeuroSense Therapeutics’ Lead Drug Candidate, PrimeC – NeuroSense Therapeutics (NASDAQ:NRSN)

Tauopathy Transcriptomes Tell Tantalizing Tales

18 Oct 2023 Fibrillar tau accumulates in several neurodegenerative diseases. Are the same molecular mechanisms responsible, or does each tauopathy follow distinct pathways? Some of both, according to a preprint posted to bioRxiv on September 30. Researchers led by Jessica Rexach and Daniel Geschwind at the University of California, Los…

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BioArctic AB – New data from LEQEMBI phase 3 Clarity AD study and subcutaneous formulation to be presented at CTAD -October 17, 2023 at 09:05 am EDT

Stockholm – BioArctic AB’s (publ) (Nasdaq Stockholm: BIOA B) partner Eisai will present new data from the phase 3 Clarity AD study for its Alzheimer’s disease (AD) treatment LEQEMBI (lecanemab-irmb) 100 mg/mL injection for intravenous use, as well as new data on the subcutaneous formulation in development at the 16th…

Continue Reading BioArctic AB – New data from LEQEMBI phase 3 Clarity AD study and subcutaneous formulation to be presented at CTAD -October 17, 2023 at 09:05 am EDT

Mitigating a TDP-43 proteinopathy by concentrating on ataxin-2 utilizing

Concentrating on ataxin-2 with RfxCas13d Due to its potential to affect TDP-43-associated toxicity20,25,26, ataxin-2 has emerged as a doubtlessly broadly relevant goal for ALS-FTD, as TDP-43 pathology is noticed in ~97% of ALS instances1,4,5 and ~45% FTD occurrences1,2,4. Given this, we sought to find out if RNA-targeting CRISPR effector proteins…

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PrimeC boosts survival of motor neurons derived from ALS patients | Potential oral therapy in Phase 2b trial with results likely this year

PrimeC, NeuroSense Therapeutics‘ investigational, fixed-dose combination therapy for amyotrophic lateral sclerosis (ALS), significantly boosted the survival of patient-derived motor neurons to levels similar to healthy controls in a lab study. The independent study was led by Justin Ichida, PhD, a professor of stem cell biology and regenerative medicine at the…

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NeuroSense’s PrimeC Shows Compelling Results for ALS Survival in Novel iPSC Model

Key Points: Groundbreaking study utilized iPSCs derived from blood samples of ALS patients to evaluate the survival rate of induced motor neurons when treated with NeuroSense’s product candidate, PrimeC. In collaboration with the University of Southern California’s Ichida Stem Cell Lab, PrimeC significantly increased the survival rate of induced motor…

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The activation of cGAS-STING in acute kidney injury

Introduction AKI, which is characterised by necroinflammation due to damage and necrosis of the kidney’s intrinsic cells,1 is a common clinical syndrome, occurring in 10–15% of hospitalized patients, whereas in the ICU this percentage is as high as 50%.2 The development of AKI is involved in the production of reactive…

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Methods for Electroporation and Transformation Confirmation in Limosilactobacillus reuteri DSM20016 (Translated to Dutch)

L.Reuteri, en specifiek de stam, DSM20016 is een robuuste, natuurlijke menselijke darm commensaal, met grote belofte voor therapeutische levering en ziektedetectie in het hele maagdarmkanaal. Dit werk zal helpen DSM20016 te positioneren als een synthetisch biologisch chassis voor dat soort toepassingen. Lactobacillen hebben geen specifiek modelorganisme. Voorkeurssoorten bestaan wel, maar…

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Understanding AMX0035’s Potential in Progressive Supranuclear Palsy

Episode 99 of the NeurologyLive® Mind Moments® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service. The Mind Moments podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including…

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NeuroSense’s PrimeC Demonstrates Outstanding Effect on ALS Survival in Innovative iPSC Model

In collaboration with the University of Southern California’s Ichida Stem Cell Lab, PrimeC was shown to significantly increase survival rate of induced motor neurons in an in vitro study utilizing induced pluripotent stem cells (iPSCs) generated from people living with ALS Results reinforce previous findings in multiple models  In a previous…

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Get It Recruit – Healthcare hiring Bioinformatics Analyst II – Remote | WFH in North Chicago, IL

Join our international team of passionate scientists, where your skills will make a meaningful impact on the future of neuroscience. Project Objective And Impact Harmonizing internal OMICS data sets, reformatting, and curating metadata is vital for enabling effective data storage according to the FAIR data principles (Findable, Accessible, Interoperable, and…

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Five impactful biotech companies to know in New York

New York – the place where people of all backgrounds come with hopes and dreams of making a better life for themselves – is just recently becoming a life sciences powerhouse, attracting many biotech companies, after a $1 billion public investment in the industry from the City of New York. …

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Creating Efficiencies in Engineered Cell Therapy Development with Comprehensive Electroporation Support

As cell therapies and other advanced modalities continue to evolve, there is increased recognition of the need to move beyond viral vehicles for the delivery of genetic cargoes into cells. While electroporation has been a mainstay in cell and genetic engineering laboratories for decades, recent advances in efficiency, reproducibility, and…

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CNM-Au8 Shows Significant Long-Term Survival Improvement in HEALEY ALS Platform Trial

Newly announced long-term follow-up data from Regimen C of the HEALEY ALS platform trial (NCT04297683) assessing the investigational agent CNM-Au8 (Clene Nanomedicine) in patients with amyotrophic lateral sclerosis (ALS) showed a decreased risk of mortality for up to 133 weeks in treated patients compared with placebo-treated patients from the Pooled…

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CT lab’s gene sequencing test finds more rare diseases

There are more than 1,200 rare disorders, and many cannot be diagnosed in a doctor’s office. That’s why genetic sequencing is so important. Now The Jackson Laboratory is able to sequence the entire genome, an advance that will roughly double the number of rare diseases that can be positively diagnosed….

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Key Details of Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy: Gunter Hoglinger, MD

WATCH TIME: 4 minutes “The most important aspects when choosing patients for this trial is to have specificity for the underlying neurobiology. We know that possible and probably patients with Richardson syndrome, as diagnosed by the MDS criteria, have a high specificity of more than 90% to have an underlying…

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Respiratory Syncytial Virus Pipeline, Clinical Trials Assessment, FDA Approvals 2023 (Updated)

PRESS RELEASE Published September 21, 2023 DelveInsight’s, “Respiratory Syncytial Virus Pipeline Insight 2023” report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Respiratory Syncytial Virus pipeline landscape. It covers the Respiratory Syncytial Virus pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Respiratory…

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AlphaMissense: DeepMind’s New AI Tool Sheds Light on Genetic Mutations

Google’s DeepMind has unveiled a new artificial intelligence tool, AlphaMissense, capable of predicting the potential harm of millions of genetic mutations. This tool aims to expedite research and enhance the diagnosis of rare disorders. Specifically, AlphaMissense focuses on missense mutations, which involve a single letter alteration in the DNA code….

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Google AI identifies millions of protein mutations capable of causing disease | Science

It’s the holy grail of modern medicine: identifying the alterations in the genome that lead to genetic diseases. It’s no easy task: each individual has thousands of mutations in the genetic information they inherited from their parents. Most are benign, but there is a percentage that can be pathogenic. Now,…

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European Medicines Agency Grants Neurosense SME Sta

(MENAFN– PR Newswire) SME staoffers regulatory guidance and engagement in dialogue with EMA NeuroSense to open an EU office in Ulm, Germany Company plans to enroll patients at multiple sites across Europe in its planned Phase 3 pivotal ALS study of PrimeC CAMBRIDGE, Mass., Sept. 20, 2023 /PRNewswire/ — NeuroSense…

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The mysterious role of ancient viruses embedded in human DNA | Health

In the blueprint for life that is the human genome, there are some mysterious, silent particles. They perform no function and have gone unnoticed for thousands and thousands of years. They are species of ancestral viruses, remnants of ancient pandemics, that became embedded in the DNA of germ cells (that…

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Widespread loss of the silencing epigenetic mark H3K9me3 in astrocytes and neurons along with hippocampal-dependent cognitive impairment in C9orf72 BAC transgenic mice.

Abstract Abstract Background Hexanucleotide repeat expansions of the G4C2 motif in a non-coding region of the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Tissues from C9ALS/FTD patients and from mouse models of ALS show RNA foci, dipeptide-repeat proteins, and notably,…

Continue Reading Widespread loss of the silencing epigenetic mark H3K9me3 in astrocytes and neurons along with hippocampal-dependent cognitive impairment in C9orf72 BAC transgenic mice.

Genetic Testing and Counseling Guidelines for ALS Promote Personalized Treatment

Credit: KATERYNA KON/SCIENCE PHOTO LIBRARY/ Getty Images Evidence-based, consensus guidelines for genetic testing and counseling for patients with amyotrophic lateral sclerosis (ALS) have been created. The 35 guideline statements are published in Annals of Clinical and Translational Neurology and include the new recommendation that all persons with ALS be offered comprehensive…

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Five biotech companies taking Los Angeles by storm

Known throughout history for being home to the iconic scene of Hollywood, in recent years Los Angeles has also become a thriving biotech hub, after years of playing second fiddle in the U.S. to the likes of South San Francisco and Boston. In this article, we take a look at…

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Transgene-free genome editing of vegetatively propagated and perennial plant species in the T0 generation via a co-editing strategy

Zhang, Y. et al. Efficient and transgene-free genome editing in wheat through transient expression of CRISPR/Cas9 DNA or RNA. Nat. Commun. 7, 12617 (2016). Article  CAS  PubMed  PubMed Central  Google Scholar  Woo, J. W. et al. DNA-free genome editing in plants with preassembled CRISPR–Cas9 ribonucleoproteins. Nat. Biotechnol. 33, 1162–1164 (2015)….

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Ultomiris failed to show benefit in ALS in clinical trial, per full data | While approved for other rare diseases, therapy did not slow ALS

Treatment with Ultomiris (ravulizumab-cwvz) — a therapy approved for several rare diseases that works by blocking activation of part of the immune system called the complement cascade — failed to slow the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial that had been launched in 2020,…

Continue Reading Ultomiris failed to show benefit in ALS in clinical trial, per full data | While approved for other rare diseases, therapy did not slow ALS

iPSC Technology Grows Even More Powerful and Disruptive

Since the discovery and development of induced pluripotent stem cell (iPSC or iPS cell) technology a decade ago, significant progress has been made within stem cell biology and regenerative medicine. In recent years, iPSCs have been used extensively in disease modeling, drug discovery, and cell therapy applications. Importantly, new pathological…

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CNM-Au8 Improves Survival in ALS, New Parkinson Risk Factor Identified, Pamrevlumab Falls Short in Phase 3 Study

WATCH TIME: 3 minutes Welcome to this special edition of Neurology News Network. I’m Marco Meglio. New data from the ongoing phase 2 RESCUE-ALS open-label extension (OLE) trial investigating CNM-Au8 (Clene Nanomedicine) demonstrated survival improvement and improved time to clinical worsening among patients with ALS treated with the agent. These…

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Alleviating symptoms of neurodegenerative disorders by astrocyte-specific overexpression of TMEM164 in mice

Liddelow, S. A. & Barres, B. A. Reactive astrocytes: production, function, and therapeutic potential. Immunity 46, 957–967 (2017). Article  CAS  PubMed  Google Scholar  Clarke, L. E. & Barres, B. A. Emerging roles of astrocytes in neural circuit development. Nat. Rev. Neurosci. 14, 311–321 (2013). Article  CAS  PubMed  PubMed Central  Google…

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This biotech is turning snake venom crotoxin into a cancer therapy

Snake venom is probably not the first thing that springs to mind when thinking about treatments or therapies, but the potential biotechnological and biomedical applications of animal venom components are actually widely recognized in scientific research. Now, Dublin-based startup Celtic Biotech is harnessing the potential of snake venom in an…

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Propulsion of Amyotrophic Lateral Sclerosis Clinical Trial Pipeline as Novel and Extensive 100+ Therapies Likely to Enter in the Domain

PRESS RELEASE Published August 29, 2023 (Albany, United States) As per DelveInsight’s assessment, globally, the Amyotrophic Lateral Sclerosis Pipeline constitutes 90+ key companies continuously working towards developing 100+ Amyotrophic Lateral Sclerosis Pipeline treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analysis DelveInsight.  …

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Identification of donor Bacteroides vulgatus genes encoding proteins that correlate with early colonization following fecal transplant of patients with recurrent Clostridium difficile

Fischer, M. et al. Fecal microbiota transplant in severe and severe-complicated Clostridium difficile: A promising treatment approach. Gut microbes 8, 289–302 (2017). Article  CAS  PubMed  Google Scholar  Van Nood, E. et al. Duodenal infusion of donor feces for recurrent Clostridium difficile. N. Engl. J. Med. 368, 407–415 (2013). Article  PubMed …

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Aldevron, Jennifer Doudna, and Innovative Genomics Institute Collaborate on In Vivo CRISPR Therapies for Neurologic Diseases

Resulting paper published in Molecular Therapy FARGO, N.D., Aug. 29, 2023 /PRNewswire/ — Aldevron, a leading global manufacturer of DNA, RNA and proteins used in cell and gene therapies and vaccine development, contributed to a publication in collaboration with 2020 Nobel Laureate Jennifer Doudna and the Innovative Genomics Institute (IGI)…

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New Grants Aim to Speed Up ALS Diagnosis Time

The ALS Association and ALS Finding a Cure have awarded $2 million in grants to help scientists worldwide find ways to shorten the time it takes to diagnose amyotrophic lateral sclerosis (ALS). Currently, it can take up to 16 months from the onset of symptoms before most individuals receive an…

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Translation dysregulation in neurodegenerative diseases: a focus on ALS | Molecular Neurodegeneration

Tandan R, Bradley WG. Amyotrophic lateral sclerosis: part 1. Clinical features, pathology, and ethical issues in management. Annals of Neurology: Official Journal of the American Neurological Association and the Child Neurology Society. 1985;18:271–80. Article  CAS  Google Scholar  Mehta P et al. Prevalence of amyotrophic lateral sclerosis (ALS), United States, 2016….

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Reference LINE-1 insertion polymorphisms correlate with Parkinson’s disease progression and differential transcript expression in the PPMI cohort

Raiz, J. et al. The non-autonomous retrotransposon SVA is trans -mobilized by the human LINE-1 protein machinery. Nucl. Acids Res. 40, 1666–1683 (2012). Article  CAS  PubMed  Google Scholar  Kim, S., Cho, C.-S., Han, K. & Lee, J. Structural variation of Alu element and human disease. Genom. Inform. 14, 70 (2016)….

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Diffuse Large B-cell Lymphoma (DLBCL) Pipeline Assessment,

(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Diffuse Large B-cell Lymphoma pipeline constitutes 80+ key companies continuously working towards developing 80+ Diffuse Large B-cell Lymphoma treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight. “Diffuse Large B-cell Lymphoma Pipeline…

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Bioinformatics Postdoctoral Fellow (SIBYLS Beamline) in Berkeley, for Lawrence Berkeley National Laboratory

Berkeley Lab’s (LBNL) Molecular Biophysics and Integrated Bioimaging Division (MBIB) has an opening for a Postdoctoral Fellow to join the SIBYLS Beamline group. In this role, you will work in a multi- disciplinary team of scientists to integrate experimental biophysical databases with machine learning algorithms. The SIBYLS Beamline is a…

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With Age, Microglia Pump cGAS, Revving Harmful Inflammation

18 Aug 2023 As people age, chronic inflammation can ensue. What drives this so-called “inflammaging”? A study published August 2 in Nature may have identified a primary cause, at least in the mouse brain. Scientists led by Andrea Ablasser at the Swiss Federal Institute of Technology in Lausanne, report that…

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In ALS, early Radicava lowers risk of ventilation, hospitalization, mortality

Early treatment with Radicava (edaravone), an approved therapy for amyotrophic lateral sclerosis (ALS), significantly reduced the risk of hospitalization, need for ventilatory support, or death compared with delaying treatment for six months. That’s according to analyses of data from Study 19 (NCT01492686), the Phase 3 clinical trial that supported Radicava’s…

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SALL1 enforces microglia-specific DNA binding and function of SMADs to establish microglia identity

Fig. 2. EKO microglia exhibit a loss of microglia identity and an increased signature of… Fig. 2. EKO microglia exhibit a loss of microglia identity and an increased signature of aging and inflammation. a, MA plot of RNA-seq data comparing WT and EKO microglia. n = 3 per group. DEGs (DESeq2 analysis…

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Ionis Pharmaceuticals, Inc. Beats Earnings Expectations in Q2 2023

Ionis Pharmaceuticals, Inc. has announced its second-quarter 2023 financial results, surpassing earnings expectations. The reported earnings per share (EPS) is $-0.6, while expectations were $-0.94. The company’s leadership team, including the Chief Executive Officer, Chief Development Officer, and Chief Financial Officer, discussed the financial results during a conference call. During…

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Bioinformatics Associate, Landau Lab – New York

The Landau Lab at the New York Genome Center is looking for a highly motivated, detail-oriented individual to join our efforts to study the basic principles of cancer evolution. As a Bioinformatics Associate, you will be trained in the analysis and interpretation of next-generation sequencing (NGS) data, originating from patient…

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