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Category: biotech/medical – Page 106

Anti-CD19 CAR T-Cell Therapy in Advanced Stiff-Person Syndrome and Concomitant Myasthenia Gravis

The immune system works to identify and target invading pathogens. Specifically, our bodies work to get rid of any harmful infections by employing a two-part immune response. The first wave of immunity is the innate immune system. This initial reaction is broad and non-specific with innate cells circulating throughout the body to detect foreign pathogens. These cells that are involved include neutrophils, macrophages, eosinophils, basophils, and dendritic cells. Once cells detect an issue, they alert the rest of the body to completely filter out the infection. Importantly, the second wave of immunity, or the adaptive immune system, elicits a strong, specific response that target pathogens the innate immune system cannot neutralize.

Adaptive immunity builds to generate robust protection against aggressive diseases. The cells that make up this response include B and T cells. B cells are mainly responsible for generating antibodies to neutralize and signal infections throughout the body. T cells are the drivers that get rid of disease. T cell activity destroys infected cells and other pathogens lingering throughout the body or site of infection. The adaptive immune response is also critical for immune memory. Once someone experiences a disease and recovers, adaptive immune cells will remember that pathogen next time it enters the body — this is how vaccines work. A patient is injected with a non-harmful virus to expose the immune system. Immediately, the body will respond and destroy the virus. However, a few T cells will also be generated to targeted similar viruses in the future. As a result, when a patient is exposed to the infection again, they will be protected and not experience symptoms.

T cells are critical for any disease or infection, including cancer. Many immunotherapies currently being develop involve activating and directing T cells to the site of the tumor. However, immunotherapies have limited efficacy due to various mechanisms around the tumor that suppress immunity. Scientists are working to understand T cell biology to develop better immunotherapies and more accurately predict treatment outcomes in patients.

Successful experiments reprogram rogue T cells for targeted autoimmune disease therapy

Two teams of researchers have developed a cell reprogramming technology that converts rogue disease-causing T cells from our immune system into protective Treg cells. These cells help ensure that the immune system doesn’t attack the body’s own tissues. The breakthroughs could usher in more personalized and targeted cell therapies for a host of autoimmune diseases.

In the first paper, published in the journal Science Translational Medicine, scientists developed a targeted cell therapy against pemphigus vulgaris (PV). This severe autoimmune skin disease causes blisters and sores.

They took the cells that were causing the disease (Dsg3-specific pathogenic T cells) from mouse models and and converted them into harmless Treg cells. They used specialized chemical tools to switch on a gene called Foxp3, which controls a cell’s ability to help the , and cut off a specific activation signal to prevent the cells from turning back into attackers.

Nanoparticle blueprints reveal path to smarter medicines

Lipid nanoparticles (LNPs) are the delivery vehicles of modern medicine, carrying cancer drugs, gene therapies and vaccines into cells. Until recently, many scientists assumed that all LNPs followed more or less the same blueprint, like a fleet of trucks built from the same design.

Now, in Nature Biotechnology, researchers from the University of Pennsylvania, Brookhaven National Laboratory and Waters Corporation have characterized the shape and structure of LNPs in unprecedented detail, revealing that the particles come in a surprising variety of configurations.

That variety isn’t just cosmetic: As the researchers found, a particle’s internal shape and structure correlates with how well it delivers therapeutic cargo to a particular destination.

Algorithm maps genetic connection between Alzheimer’s and specific neurons

The number of people living with dementia worldwide was estimated at 57 million in 2021 with nearly 10 million new cases recorded each year. In the U.S., dementia impacts more than 6 million lives, and the number of new cases is expected to double over the next few decades, according to a 2025 study. Despite advancements in the field, a full understanding of disease-causing mechanisms is still lacking.

To address this gap, Rice University researchers and collaborators at Boston University have developed a that can help identify which specific types of cells in the body are genetically linked to complex human traits and diseases, including in forms of dementia such as Alzheimer’s and Parkinson’s.

Known as “Single-cell Expression Integration System for Mapping genetically implicated Cell types,” or seismic, the tool helped the team hone in on genetic vulnerabilities in memory-making brain cells that link them to Alzheimer’s ⎯ the first to establish an association based on a genetic link between the disease and these specific neurons. The algorithm outperforms existing tools for identifying that are potentially relevant in complex diseases and is applicable in disease contexts beyond dementia.

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