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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease 142
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Patient-derived organoids in disease modelling

Drug Target Review

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

Disease 122
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Discovery of 'new rules of the immune system' could improve treatment of inflammatory diseases, say scientists

Science Daily: Pharmacology News

Tests, in mice, of a drug developed by the researchers showed that regulatory T cells can be attracted to specific body parts, boosted in number, and activated to suppress immune response and rebuild tissue.

Treatment 118
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Scientists discover a cause of lupus and a possible way to reverse it

Science Daily: Pharmacology News

Scientists have discovered a molecular defect that promotes the pathologic immune response in systemic lupus erythematosus (known as lupus) and show that reversing this defect may potentially reverse the disease.

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Using next-gen CRISPR tool, scientists create unprecedented molecular map of human immune response

Science Daily: Pharmacology News

Their findings uncover detailed information that could help overcome the limitations of today's immunotherapies and identify new drug targets for a wide range of diseases, including autoimmune diseases and cancer.

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Rare autoantibody diseases: an innovative targeted pathway

Drug Target Review

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. Antibodies protect us by attaching to molecules on those invaders (known as antigens) and triggering our body’s natural immune response to destroy them.

Disease 98
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Study reveals how some bacterial infections become chronic

Broad Institute

By examining the genomes of bacteria collected from hundreds of people with persistent Salmonella infections, they discovered genetic mutations that both reduce the bacteria’s “virulence,” or ability to infect, and dampen the host’s immune responses, creating a kind of molecular camouflage that shields the bacteria from the immune system’s gaze.