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Category: biotech/medical – Page 528

Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy

Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy. This is the first human trial of gene supplementation therapy targeting this condition.

Retinal dystrophy caused by biallelic variants in the AIPL1 gene leads to severe visual impairment from birth, with progressive degeneration and limited treatment options. Previous studies of early-onset rod-cone dystrophies, including AIPL1-related forms, highlighted a critical window for intervention during early childhood, when some photoreceptor structure remains intact. Prior research using Aipl1-deficient mouse models and human retinal organoids demonstrated partial restoration of photoreceptor function through .

In the study, “Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study,” published in The Lancet, researchers administered a single subretinal injection of a recombinant adeno-associated (rAAV8.hRKp. AIPL1) carrying the AIPL1 gene to one eye of each child to assess the safety and efficacy of gene supplementation therapy in improving visual function and preserving retinal structure.

Schizophrenia is reflected in the brain structure, study shows

The symptoms of schizophrenia vary greatly from person to person. A new study appearing in the American Journal of Psychiatry shows how these differences manifest themselves in the structure of the brain.

Schizophrenia is a complex mental health condition that affects perception, thought and emotions. This complexity is reflected in the individual manifestations of the disease: for some patients, perceptual disturbances are the main problem, while for others, cognitive impairments are more prevalent.

“In this sense, there is not one , but many, each with different neurobiological profiles,” says Wolfgang Omlor, first author of the study and senior physician at the University Hospital of Psychiatry Zurich.

ErythroMer and Beyond: The Future of Blood Technology

What if a simple vial of synthetic blood could save millions of lives? From cutting-edge laboratories to the frontlines of disaster zones, scientists are revolutionizing medicine with the development of artificial blood. In this episode of Beyond the Veil, we take you on a journey into the world of groundbreaking innovations like ErythroMer, a shelf-stable synthetic red blood cell, and lab-grown blood cells that could transform healthcare as we know it.

Join us as we explore:
🔬 The intricate science behind replicating blood\’s vital functions.
💡 How Dr. Allan Doctor and his team are making synthetic red blood cells a reality with ErythroMer.
🩸 The NHS’s revolutionary trial of lab-grown red blood cells.
🌍 The potential to save lives in remote areas, on battlefields, and even during space exploration.
🚀 The hurdles, breakthroughs, and future implications of artificial blood research.

💉 This isn\’t just a story about science; it\’s a tale of perseverance, hope, and the determination to rewrite the rules of medicine.

Sources and Research Cited in this Episode:
📖 The Long Quest for Artificial Blood — New Yorker.
🔗 https://www.newyorker.com/magazine/2025/02… Challenges in Hemoglobin-Based Oxygen Carriers — LabMed 🔗 https://www.labmed.theclinics.com/article/.… 👉 Subscribe to Beyond the Veil for more deep dives into the mysteries of science, medicine, and technology. Together, let’s explore what lies beyond the edge of discovery. #ArtificialBlood #SyntheticBlood #MedicalInnovation #BeyondTheVeil.

📖 Lab-Grown Red Blood Cells Clinical Trial (RESTORE) — NHS Blood and Transplant.
🔗https://www.newyorker.com/magazine/2025/02…

Automatic cell analysis: AI-powered software ‘segments anything’ in microscopy images

Identifying and delineating cell structures in microscopy images is crucial for understanding the complex processes of life. This task is called “segmentation” and it enables a range of applications, such as analyzing the reaction of cells to drug treatments, or comparing cell structures in different genotypes.

It was already possible to carry out automatic segmentation of those biological structures, but the dedicated methods only worked in specific conditions and adapting them to new conditions was costly. An international research team led by Göttingen University has now developed a method for retraining the existing AI-based software Segment Anything on over 17,000 with over 2 million structures annotated by hand.

The new model is called Segment Anything for Microscopy and it can precisely segment images of tissues, cells and similar structures in a wide range of settings. To make it available to researchers and medical doctors, they have also created μSAM, user-friendly software to “segment anything” in microscopy images. The work is published in Nature Methods.

Blebbisomes are large, organelle-rich extracellular vesicles with cell-like properties

Super cool paper where Jeppesen et al. discover and characterize a new type of large extracellular vesicle (EV) that they call blebbisomes! These blebbisomes have active mitochondria as well as other organelles (except nucleus), secrete and take up smaller EVs, and can reach sizes of up to 20 micrometers! #cellbiology #molecularbiology #biochemistry

Cells release a variety of 30-to 10,000-nm lipid-bilayer-enclosed extracellular vesicles (EVs) to facilitate cell-to-cell and cell-to-environment communication by packaging signalling molecules to avoid degradation1,2,3,4,5 and escape immune surveillance6,7,8,9. EVs may interact with target cells through contact between molecules on the EV surface with receptors on the cell surface to relay signals. In addition, modulation of recipient cell behavior may follow uptake of EVs cargo, including bioactive proteins, lipids and nucleic acids. EVs have emerged as important actors and agents of intercellular communication in normal cell biology and pathological conditions2,4,6.

Here, we identify blebbisomes, an exceptionally large functional EVs, that are actively released by human and mouse cells, remain motile independently of cells and have the capacity to both take up EVs and secrete exosomes and microvesicles. Blebbisomes are the largest type of EV described so far with an average diameter of 10 µm but can be as large as 20 µm, with an area commonly larger than 50 µm2. After being released from motile cells, blebbisomes display marked contractility-dependent ‘blebbing’ behaviour. Both normal and cancer cells release blebbisomes that contain active, healthy, mitochondria further distinguishing them from other large EVs (lEVs) such as exophers10,11 and migrasomes12 that function in the removal of damaged mitochondria from cells under stress conditions. In addition, blebbisomes contain many other cellular organelles including endoplasmic reticulum (ER), Golgi apparatus, ribosomes, lysosomes, endosomes, multivesicular endosomes (MVEs) and autophagosomes/amphisomes, as well as cytoskeletal elements; however, they lack a definable nucleus.

Herpesviruses may contribute to Alzheimer’s disease via transposable elements

Researchers from Cleveland Clinic’s Genome Center have outlined the pathway human herpes simplex virus-1 (HSV1) can use to contribute to Alzheimer’s disease in aging brains. In a report published in Alzheimer’s & Dementia, investigators also share two FDA-approved, commercially available drugs that reverse this pathway in a laboratory setting.

The findings are the first concrete evidence to support the previously controversial link between human herpesviruses (HHVs) and Alzheimer’s disease. Illustrating the potential for herpes to trigger dementia aids continued efforts to prevent and cure neurodegenerative disease, says senior author and Genome Center director Feixiong Cheng, Ph.D…

For most people, contracting a is just an inconvenient or harmless fact of life. Many herpesviruses are individually present in a large percentage of people worldwide, meaning virtually every human being on earth is expected to contract at least three types of herpesviruses by adulthood. Some of these viruses don’t cause symptoms, while others only cause minor illnesses like mono or chickenpox. However, even after these illnesses subside, an infected individual still carries herpesviruses for the rest of their life, with only minor symptoms like occasional cold sores.

Dysfunctional CD11c−CD21− extrafollicular memory B cells are enriched in the periphery and tumors of patients with cancer

New findings cast light on the role of understudied DN3 memory B cells in cancer, and shows these cells correlate with progression and poor clinical outcomes in head and neck squamous cell carcinoma and melanoma.

📄

Tumor-associated extrafollicular B cells are phenotypically, locationally, and functionally distinct from germinal center–dependent memory B cells.

Hidden Brain Lesions Uncovered by AI, Transforming Epilepsy Treatment

An AI-powered tool called MELD Graph is revolutionizing epilepsy care by detecting subtle brain abnormalities that radiologists often miss.

By analyzing global MRI data, the tool improves diagnosis speed, increases access to surgical treatment, and cuts healthcare costs. Though not yet in clinical use, it is already helping doctors identify operable lesions, offering hope to epilepsy patients worldwide.

AI Breakthrough in Epilepsy Detection.

Scientists invent new drug candidates to treat antibiotic-resistant bacteria

There’s an arms race in medicine—scientists design drugs to treat lethal bacterial infections, but bacteria can evolve defenses to those drugs, sending the researchers back to square one. In an article published in the Journal of the American Chemical Society, a University of California, Irvine-led team describes the development of a drug candidate that can stop bacteria before they have a chance to cause harm.

“The issue with antibiotics is this crisis of antibiotic resistance,” said Sophia Padilla, a Ph.D. candidate in chemistry and lead author of the new study. “When it comes to antibiotics, can evolve defenses against them—they’re becoming stronger and always getting better at protecting themselves.”

About 35,000 people in the U.S. die each year from from pathogens like Staphylococcus, while about 2.8 million people suffer from bacteria-related illnesses.

Engineers show industrial potential of diamond quantum sensors for EV battery monitoring

Researchers at the Technical University of Munich (TUM) have invented an entirely new field of microscopy called nuclear spin microscopy. The team can visualize magnetic signals of nuclear magnetic resonance with a microscope. Quantum sensors convert the signals into light, enabling extremely high-resolution optical imaging.

Magnetic resonance imaging (MRI) scanners are known for their ability to look deep into the human body and create images of organs and tissues. The new method, published in the journal Nature Communications, extends this technique to the realm of microscopic detail.

“The used make it possible to convert signals into optical signals. These signals are captured by a camera and displayed as images,” explains Dominik Bucher, Professor of Quantum Sensing and researcher at the Cluster of Excellence Munich Center for Quantum Science and Technology (MCQST).

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