Background: We previously demonstrated the successful use of in vivo CRISPR gene editing to delete 4-hydroxyphenylpyruvate dioxygenase (HPD) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hereditary tyrosinemia type 1 (HT1). The aim of this study was to develop an ex vivo gene-editing protocol and apply it as a cell therapy for HT1. Methods: We isolated hepatocytes from wild-type (C57BL/6J) and Fah-/- mice and then used an optimized electroporation protocol to deliver Hpd-targeting CRISPR-Cas9 ribonucleoproteins into hepatocytes. Next, hepatocytes were transiently incubated in ...

University of Georgia-based startup CyanVac LLC received federal funding to support a comparative Phase 2b clinical trial of CVXGA, the company’s intranasal vaccine candidates designed to protect against COVID-19. As part of the award from Project NextGen, a federal initiative based in the U.S. Department of Health and Human Services (HHS), CyanVac will sponsor a randomized, double-blind Phase 2b study with 10,000 participants to compare the efficacy and safety of the intranasal vaccine against an FDA-approved mRNA-based COVID-19 vaccine. The new vaccine is based on a viral delivery platform developed by He containing modified ...

Patients are less likely to fill prescriptions for naloxone when they face increases in out-of-pocket costs, according to U-M researchers. Patients are less likely to fill prescriptions for naloxone when they face increases in out-of-pocket costs, according to research by the University of Michigan. Published in the Journal of the American Medical Association, the study utilized data from a national pharmacy transactions database from November 2020 to March 2021. Researchers found that about 1 in 3 naloxone ...

New research by Swansea University and the University of Zurich has found that sharks retained high levels of functional diversity for most of the last 66 million years, before steadily declining over the last 10 million years to its lowest value in the present day. Modern sharks are among the ocean’s most threatened species; yet have notably survived numerous environmental changes in their 250-million-year history. Today, their more than 500 species play many different ecological roles, from apex predators to nutrient transporters. Ecological roles are determined by species’ traits such as body size, ...

Periods of fasting reprogram the immune system’s natural killer cells to better fight cancer, according to a new study in mice from researchers at Memorial Sloan Kettering Cancer Center (MSK). Fasting and other dietary regimens are increasingly being explored as ways to starve cancer cells of the nutrients they need to grow and to make cancer treatments more effective. Now a team of researchers from MSK’s Sloan Kettering Institute and their collaborators have shown for the ...

Sjögren’s disease is a systemic autoimmune disorder that often causes sicca symptoms – or dryness of the eyes and mouth, alongside many other complications such as fatigue, pain, and neurological manifestations. There are treatments that help with symptoms, but none that address the underlying disease processes.3 One key issue in developing new treatments is that Sjögren’s can differ from person to person – and that makes it hard to measure and compare outcomes in a clinical trial.   NECESSITY is an interventional trial looking at new clinical endpoints in Sjögren’s disease. ...

Inflammatory arthritis describes a group of diseases caused by an overactive immune system. The different types of inflammatory arthritis include rheumatoid arthritis, psoriatic arthritis, juvenile idiopathic arthritis, and ankylosing spondylitis. The most common symptoms are joint pain and stiffness. Pain is the predominant symptom in the majority of people with inflammatory arthritis, which contributes to the global burden of rheumatic and musculoskeletal conditions.1 Knowledgeable support can reduce ...

Autologous hematopoietic stem cell transplantation (aHSCT) for SSc has been proven the most effective treatment strategy with regard to overall and event free survival in selected patients.2 But a key limitation is its toxicity, and new treatment options are needed. Two abstracts presented at the 2024 EULAR congress in Vienna focused on novel approaches.   Jörg Henes presented on behalf of the AST MOMA investigators. This prospective, open-label, study evaluated the feasibility of aHSCT in patients with impaired lung or heart function, and also ...

BMO in the sacroiliac joint on MRI is present in up to 84% of people with non-radiographic axSpA – but it is also often seen in a non-inflammatory setting, such as in women after childbirth.1,2 As back pain is common after childbirth, differential diagnosis with axSpA is an important issue in clinical practice.1 In axSpA, active inflammatory lesions are likely to progress to structural lesions over time – particularly fat lesions and erosions. But it is not known whether the same is true for BMO induced by mechanical stress. In June 2024, EULAR – The European Alliance of Associations for Rheumatology – held its annual congress ...

In 2019, a Swedish prospective birth cohort study of over 15,000 children showed that consuming fish at least once a week during pregnancy and during the first year of life was associated with up to a 5-fold increased risk of JIA, compared to those with fish consumption less than once a week. This increased risk was primarily attributed to elevated exposure to heavy metals.1 Now, new research shared at the 2024 congress of EULAR – The European Alliance of Associations for Rheumatology – is investigating ...

SSc is a connective tissue disease with variable clinical presentation. It may affect the skin, blood vessels, heart, lungs, kidneys, gastrointestinal tract, and musculoskeletal system – and this complexity and diversity makes it challenging to treat.2 This clinical heterogeneity in SSc may be partially explained by SSc-specific antibodies, but a better understanding of additional risk factors and patient stratification is still needed. Three abstracts shared at the 2024 EULAR congress present a selection of new clinical research to enrich knowledge ...

In an animal model of Duchenne muscular dystrophy, Brazilian researchers tested a therapy that combines photobiomodulation using laser light or light-emitting diodes (LEDs) with idebenone, an antioxidant compound investigated for application in neurodegenerative diseases. As reported in an article published in the journal PLOS ONE, the strategy prevented muscle degeneration and improved regenerative capacity in the muscle fibers affected by the disease. Duchenne muscular dystrophy is the most common and most severe form of childhood muscular dystrophy and most often ...

MINNEAPOLIS, June 14, 2024 — NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) announced interim results from the ACCESS trial as an oral abstract during the European Hematology Association (EHA) Annual Meeting in Madrid, Spain. The study demonstrated that adults with hematologic malignancies who received peripheral blood stem cell (PBSC) transplant from HLA-mismatched unrelated donors (MMUD) followed by post-transplant cyclophosphamide (PTCy) graft-versus-host-disease (GvHD) prophylaxis exhibited a 79% overall survival (OS), the primary ...

Hamilton, ON (June 14, 2024) – A widely available drug helps prevent upper gastrointestinal bleeding in critically ill adults on a breathing machine, according to the results of a global study and meta-analysis led by researchers at McMaster University. The research, published on June 14, 2024 in The New England Journal of Medicine and NEJM Evidence, investigated the effect of the gastric acid suppressant pantoprazole, which is primarily used to treat heartburn caused by gastroesophageal reflux disease (GERD). Patients ...

In a breakthrough that could redefine tea crop protection, a new study has shed light on the genetic makeup of the tea grey geometrid, Ectropis grisescens. Through the re-sequencing of 43 genomes, scientists have mapped out the pest's population structure and its remarkable adaptation to tea crops, offering new avenues for managing this agricultural adversary. Amidst the lush tea plantations, a microscopic menace looms—the tea grey geometrid, a pest that can decimate tea yields with its insatiable appetite. The economic and qualitative havoc wreaked by this insect has prompted an urgent call for innovative pest control solutions. However, the genomic secrets ...

About The Study: This repeated cross-sectional study found that past-year Papanicolaou testing rates were lower in 2022 than 2019, pointing to a need to increase access to screenings to prevent an uptick in cervical cancer incidence. Rural-vs-urban differences in 2022 indicate a need to specifically target rural females.  Corresponding Author: To contact the corresponding author, Tyrone F. Borders, Ph.D., email ty.borders@uky.edu. To access the embargoed study: Visit our For The Media website at this link https://media.jamanetwork.com/ (doi:10.1001/jamanetworkopen.2024.17094) Editor’s ...

    Parrots, songbirds, and hummingbirds can learn to make new sounds. No-one knew, but New Zealand’s smallest bird, the rifleman or titipounamu, may have a rudimentary version of the same talent.   University of Auckland research into the bird is part of a rethinking of how and when vocal learning evolved in birds.  Scientists traditionally assumed birds were split into two groups - those which can learn sounds (parrots, songbirds, and hummingbirds) and those which can’t - but the study published in the scientific journal Communications Biology ...

In recent years, high-entropy carbide ceramics have received extensive attention and become another research focus in the high entropy materials field, which are also known as multi-component carbide ceramics. The multi-component carbide ceramics not only inherit the special properties of high-entropy materials brought by complex compositions, but also keep the advantages of transition metal carbide ceramics as a kind of ultra-high temperature ceramics (UHTCs), such as high melting point, high-temperature stability, high Young's modulus, high hardness, and ...

Curbing the confirmed human influence on the climate system and mitigating climate change require profound transformation of global energy system. In this context, modeling technical progress and innovation within Integrated Assessment Models (IAMs) is crucial for providing insights about the consequences of long-term energy system transformation, and capturing the development of several interacting systems and technical evolution process.   A review of Endogenous Technological Change (ETC) written by Hongbo Duan ...

INDIANAPOLIS – The Indiana Sickle Cell Dashboard, launching this month on the Regenstrief Institute website, presents a dynamic, panoptic picture of sickle cell disease throughout an entire state. The new dashboard uses de-identified data from throughout the state of Indiana, obtained from multiple clinical and administrative sources, to present easy-to-understand, interactive visualizations of the disease’s prevalence and burden. The pioneering dashboard, created, developed and maintained by the Regenstrief Institute, is one of the first ...

Synthetically generated population data can reveal the equity impacts of distributing transportation resources and funding across diverse regions, according to new research from NYU's Tandon School of Engineering that uses New York State as a case study. Relying on an artificial dataset representing 19.5 million New York residents and over 120,000 modeled origin-destination trips, researchers from NYU Tandon's C2SMARTER, a Tier 1 U.S U.S. Department of Transportation-funded University Transportation Center, determined how best to invest in transportation services when equitable benefits are an objective. They ...

In a new study, published in Social Psychological and Personality Science, researchers share evidence that people’s attitudes are deeply woven into language and culture across the globe and centuries. The researchers looked at connections between people’s attitudes and language from 55 different topics like rich vs. poor, dogs vs. cats, or love vs. money. They used four text sources: Current English writing and text, English books going back 200 years, and texts in 53 languages other than English. As a measure of people’s attitudes, they used data from over 100,000 Americans; first, direct self-reports, and second, an indirect ...

Pregnant women who have been vaccinated against Covid-19 are less likely to have a caesarean section or experience hypertension, according to a study.          A meta-analysis funded by the National Institute for Health and Care Research Birmingham Biomedical Research Centre of 67 studies which included more than 1.8m women found that being fully vaccinated against COVID-19 had a protective benefit against infection and hospitalisation, while vaccination with at least one dose lowered the risk of adverse pregnancy-related and neonatal outcomes.   Drawing on ...

An artificial intelligence-powered method for detecting tumor DNA in blood has shown unprecedented sensitivity in predicting cancer recurrence, in a study led by researchers at Weill Cornell Medicine, NewYork-Presbyterian, the New York Genome Center (NYGC) and Memorial Sloan Kettering Cancer Center (MSK). The new technology has the potential to improve cancer care with the very early detection of recurrence and close monitoring of tumor response during therapy. In the study, which appears June 14 in Nature Medicine, the researchers showed that they could train a machine learning model, a type ...

Washington DC, June 14, 2024: A study by authors from the International Food Policy Research Institute (IFPRI), published today in Nature Sustainability, reaffirms the world’s growing dependence on depleting groundwater systems. Although efforts to slow down groundwater depletion need to be urgently accelerated, this study indicates that such efforts – in the absence of other accompanying measures – would likely lead to significant food security impacts. The study finds that ending groundwater depletion would lead to sharp declines in food production, ...