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Gene therapy trial offers breakthrough for rare genetic disorder

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  • 2025-07-25 20:00 event
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Gene therapy trial offers breakthrough for rare genetic disorder
A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for the genetic disorder.

1.218. Scientists advance efforts to create 'virtual cell lab' as testing ground for future research with live cells

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Using mathematical analysis of patterns of human and animal cell behavior, scientists say they have developed a computer program that mimics the behavior of such cells in any part of the body. Led by investigators at Indiana University, Johns Hopkins Medicine, the University of Maryland School of Medicine and Oregon Health & Science University, the new work was designed to advance ways of testing and predicting biological processes, drug responses and other cell dynamics before undertaking more costly experiments with live cells.

1.219. Globally, over 2.5 million COVID deaths prevented worldwide thanks to vaccines, data indicate

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Thanks to vaccinations against SARS-CoV-2 in the period 2020–2024, 2.533 million deaths were prevented at the global level; one death was avoided for every 5,400 doses of vaccine administered.

1.220. Scientists develop tissue-healing gel using milk-derived extracellular vesicles

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Researchers from Columbia Engineering have established a framework for the design of bioactive injectable hydrogels formulated with extracellular vesicles (EVs) for tissue engineering and regenerative medicine applications.

1.221. First-of-its kind study reveals psychological toll of war in Gaza extends far beyond the region

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The ongoing conflict in the Middle East is having a significant impact on the mental health and well-being of Australian women who migrated from directly impacted countries, or have family ties to the region, according to researchers from UNSW Sydney and The Australian National University (ANU).

1.222. Elite soccer players master the art of trapping without breaking their stride: Study

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In soccer games, "ball-trapping"—quickly changing direction after receiving a pass to transition into an attack—is a critical maneuver. This action can significantly influence the flow of a match, especially when executed effectively in the space between an opponent's midfield and defensive lines. However, the specific physical techniques used by skilled ball-trappers during actual gameplay have not been well understood.

1.223. Number of family caregivers has skyrocketed in the US

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The number of Americans caring for an older or disabled family member has risen dramatically during the past 10 years, according to a new AARP policy report.

1.224. Plant virus triggers immune response that targets and destroys cancer cells

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A virus that typically infects black-eyed peas is showing great promise as a low-cost, potent cancer immunotherapy—and researchers are uncovering why.

1.225. New research uncovers gene impacts of PFAS exposure in firefighters

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Researchers at the University of Arizona Mel and Enid Zuckerman College of Public Health found that certain kinds of long-lasting chemicals firefighters are exposed to may affect the activity of genes linked to cancer and other diseases. The findings appear in the journal Environmental Research.

1.226. What is chikungunya virus, and should we be worried about it in Australia?

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This week, the World Health Organization (WHO) raised concerns about a surge in the number of cases of a mosquito-borne viral infection called chikungunya.

1.227. Gene therapy trial offers breakthrough for rare genetic disorder

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A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for the genetic disorder.

1.228. Study finds key gene loss may trigger premature aging in the brain

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Age often brings a gradual decline in the ability to learn new things and retain memories. This phenomenon, often associated with the elderly, is linked to the brain's deteriorating capacity to generate new neurons—a process that primarily occurs in the hippocampus—as neural stem cells (NSCs) divide and mature.

1.229. Trial finds no protective effect of moisturizers in atopy-prone infants

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Oregon Health & Science University-led research is reporting that daily full-body emollient use starting before 9 weeks of age lowered atopic dermatitis incidence by 24 months in a representative US infant cohort.

1.230. GLP1 weight loss injections may reduce asthma symptoms

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GLP1 agonist drugs, commonly known by brand names such as Ozempic and Mounjaro, reduce asthma symptoms in obese people according to a new study from the University of Aberdeen and the Observational and Pragmatic Research Institute (OPRI), Singapore.

1.231. Researchers identify genetic marker that could guide brain cancer treatment

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University of Kentucky Markey Cancer Center researchers have discovered a genetic biomarker that could help identify patients with glioblastoma most likely to benefit from the cancer drug bevacizumab.

1.232. Hormone found to suppress immune cells, allowing cancer to evade detection

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Researchers at UT Southwestern Medical Center have discovered how a hormone interacts with a receptor on the surface of immune cells to shield cancer cells from the body's natural defenses.

1.233. Study shows DNA regulatory switch prompts stem cells to give rise to blood

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A new study led by Stephen D. Nimer, M.D., director of Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, shows how a key molecule regulates the generation of new blood cells, a process called hematopoiesis that goes awry in cancer.

1.234. Texas counties have some of the highest STD rates in US, new report says

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Sexually transmitted infections are on the rise in Texas. And in some parts of the state, the numbers are climbing higher than the national average.

1.235. Human CLOCK gene enhances brain connectivity and mental flexibility in mice, study finds

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Clock genes are a set of genes known to contribute to the regulation of the human body's internal 24-hour cycle, also known as the circadian rhythm. One of these genes is the so-called CLOCK gene, a protein that regulates the activity of other genes, contributing to recurrent patterns of sleep and wakefulness.

1.236. Measles cases just reached 30-year high in the US: How bad is California surge?

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Measles cases in California and the United States are climbing to levels the country hasn't seen in years.

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