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Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. The tools are known as enhancer AAV vectors.
4c15843 Peptide macrocycles are promising therapeutics for a variety of disease indications due to their overall metabolic stability and potential to make highly selective binding interactions with targets. Boyd, Lei Wang, Ralf Bartenschlager, and Matthew Bogyo Journal of the American Chemical Society 2025 DOI: 10.1021/jacs.4c15843
Combined with network pharmacology and experimental verification, this study found that platycodin D and luteolin in Jiegeng ( Platycodonis Radix ) may regulate inflammation through the IL-17 signaling pathway to treat influenza virus pneumonia.
Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. Viral vectors use a virus’s blueprint to deliver genetic material into cells.
There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& & The new study, published in Cell , is the first time prime editing has been used to treat a neurological disease in animals, offering hope for treating people with AHC and other genetic brain disorders.
Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers. To date, more than 160 monoclonal antibody therapies targeting nearly 100 disease-related proteins have received regulatory approval globally.
By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.
This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Dr. Octavio Ramilo, Chair of the Department of Infectious Diseases at St. A New Era in Infant RSV Protection Dr. Dean Y.
Importantly, both cases of ALF have been reported exclusively in non-ambulatory individuals with Duchenne muscular dystrophy — a population that typically bears a heavy disease burden — underscoring the need for careful and tailored approaches to their care. Sarepta’s Chief Scientific Officer and Head of Research and Development. “We
Gene editing disrupts Huntingtons mutation in mice By Allessandra DiCorato May 27, 2025 Breadcrumb Home Gene editing disrupts Huntingtons mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntingtons disease and Friedreichs ataxia.
Initially approved for dengue fever, post-market data revealed that the vaccine increased the risk of severe disease in patients who had never been exposed to the virus before vaccination. Conversely, Sanofi’s Dengvaxia vaccine case highlights the consequences of inadequate post-market surveillance.
Importantly, this will enable them to produce mRNA vaccines during periods between health crises, retaining trained personnel, technical expertise, and production capacity while retaining the ability to respond quickly in the event of future disease outbreaks.
Merck’s ENFLONSIA Receives ACIP Endorsement for RSV Prevention in Infants, Marking a Major Milestone in Pediatric Infectious Disease Prevention U.S. Understanding the Burden of RSV in Infants RSV is a common respiratory virus that typically circulates during the fall and winter months in the U.S., pediatric population. As the U.S.
1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV).
This update comes after two cases of fatal acute liver failure (ALF) were reported in non-ambulatory DMD patients who received Elevidys, a phenomenon that underscores the ongoing complexities and safety signals related to adeno-associated virus (AAV)-mediated gene therapy.
Sanofi Ramps Up Early Global Distribution of Beyfortus to Meet Growing RSV Season Demand, Enhancing Protection for Infants Worldwide In a proactive move to ensure readiness for the 2025-2026 respiratory syncytial virus (RSV) season, Sanofi has announced that shipments of Beyfortus® (nirsevimab) will begin in early Q3 2025.
Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.
By binding to the active site of the protease, the drug prevents this cleavage process, effectively halting viral assembly and impeding the virus’s ability to produce future virions. [1] Jump up to: a b “Coronavirus disease 2019 (COVID-19) emerging treatments” BMJ Best Practice US. Retrieved 27 December 2023.
A cat named Pepper has once again helped scientists discover a new virus—this time a mysterious orthoreovirus found in a shrew. Although once thought to be harmless, these viruses are increasingly linked to serious diseases in humans and animals.
“Overcoming pathways to enable mechanisms within the immune system to combat disease has already seen validation with some of the recently approved drugs in the short- and medium-term,” Galbraith explains. “This is encouraging and is likely to stimulate investment moving forward.”
2 Drug use contributes to the spread of HIV and other infectious diseases like hepatitis B and C in multiple ways, including the sharing of drug injection equipment. Another study in Baltimore found an especially strong effect of SSP usage on later drug treatment among people living with HIV. 6 SSPs also may distribute fentanyl test strips.
Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. This essay focuses on how we might do both, specifically for the cell. Subscribe to Asimov Press.
Jacob Bell/BioPharma Dive Dive Brief: The Food and Drug Administration on Thursday granted full approval for Moderna’s COVID-19 vaccine Spikevax in children aged 6 months through 11 years who are at an increased risk for COVID disease. The shot was previously available for these individuals under emergency use authorization. Kennedy Jr.
Spencer Platt via Getty Images The first marketed treatment for the insatiable hunger associated with Prader-Willi disease is selling more quickly than Wall Street analysts expected, an early, but encouraging sign of demand. The disease, which affects an estimated 10,000 to 20,000 people in the U.S.,
With this approval, MAVYRET becomes the first and only DAA treatment approved to treat both acute and chronic hepatitis C virus (HCV) infections in patients as young as three years old, with or without compensated cirrhosis, using an eight-week regimen. No one should die of hepatitis C.” Global and U.S.
One such initiative includes a collaboration with the Bill & Melinda Gates Foundation aimed at developing low-cost monoclonal antibodies (mAbs) for infectious diseases such as malaria and respiratory syncytial virus (RSV). This partnership has recently reached a key milestone, resulting in a second disbursement of $1.5
Team leader David Duffy, professor of wildlife disease genomics at the University of Florida‘s Whitney Laboratory for Marine Bioscience, was surprised at the ease of sampling and analyzing the eDNA. The approach can detect emerging infectious diseases like new flu or COVID variants, as well as track allergens.
Temporary Recommendations: Strengthened Global Coordination and National Action The revised temporary recommendations, applicable until at least 20 August 2025, target all WHO Member States experiencing mpox virus (MPXV) transmission. Collaborative Surveillance Strengthening disease surveillance remains a central pillar of the response.
WEDNESDAY, July 9, 2025 — A common virus once thought harmless to humans might be linked to Parkinson’s disease, a new study says.The germ, Human Pegivirus (HPgV), was found in half the autopsied brains of patients with Parkinson’s, but not in any b.
Marks wrote that the FDA has “taken on aspects of policymaking that have previously been in the domain” of the Centers for Disease Control and Prevention. In addition, he said the changes ignore the existing safety data for the COVID-19 shots and the continued hospitalizations and deaths caused by the virus.
Despite the fact that vaccine-preventable diseases pose significant risks across the lifespan, adult immunization remains underutilized and inconsistently prioritized. The potential returns in LMICs could be even greater, given the higher disease burden and lower baseline coverage. Adult immunization is central to that shift.”
14, 2024 -- Parvovirus B19, a seasonal respiratory virus that subsided during the pandemic, is making a comeback, U.S. Centers for Disease Control and. WEDNESDAY, Aug. health officials warned Tuesday.In a health alert issued by the U.S.
TUESDAY, July 10, 2024 -- An experimental “air mask” could help ward off infectious diseases while people are on the job, researchers report.The mask uses an air curtain blowing down from the brim of a hard hat to prevent airborne viruses from rea.
Researchers have come up with a new way to identify more infectious variants of viruses or bacteria that start spreading in humans -- including those causing flu, COVID, whooping cough and tuberculosis.
Zika virus hijacks the skin of its human host to send out chemical signals that lure more mosquitoes to infect and spread the disease further, new research shows.
The biotech has raised about $65 million to test two ways to deliver larger genes into the body, each of which could help gene therapy treat more diseases.
While we often think of diseases as caused by foreign bodies -- bacteria or viruses -- there are hundreds of diseases affecting humans that result from errors in cellular production of its proteins.
WEDNESDAY, May 8, 2024 -- The virus behind COVID has mutated again, this time producing variants nicknamed FLiRT, the U.S. Centers for Disease Control and Prevention (CDC) has reported.The variants are appearing in wastewater sampling, the CDC.
Centers for Disease Control and Prevention said that a surge in respiratory illnesses in China is not being fueled by a new. FRIDAY, Dec. 1, 2023 (Healthday News) -- In testimony provided Thursday to members of Congress, the head of the U.S.
A new study provides a list of the wildlife species present at the market from which SARS-CoV-2, the virus responsible for the COVID-19 pandemic, most likely arose in late 2019. The study is based on a new analysis of metatranscriptomic data released by the Chinese Center for Disease Control and Prevention (CDC).
3] It was discovered for the treatment of hepatitis C [4] and has since been investigated for use against other viral diseases such as AIDS and COVID-19 , [2] [5] for which it was granted conditional approval in China. [6] It was approved in China in 2021 as a first-in-class treatment for human immunodeficiency virus (HIV). Swallow, S.;
THURSDAY, June 29, 2023 -- Americans ages 60 and up can get their vaccine for respiratory syncytial virus (RSV) this fall, the U.S. Centers for Disease Control and Prevention announced Thursday. On Thursday, Dr. Rochelle Walensky, the outgoing CDC.
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