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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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T cells’ vital role in boosting immune responses

Drug Target Review

Among the many remarkable components that make up this complex defence network, T cells have emerged as powerful guardians, orchestrating an array of immune responses with great precision.

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Leveraging agonist antibodies to address immunological diseases

Drug Target Review

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies of immune checkpoint regulators These represent a groundbreaking class of immunotherapeutic agents that mimic the natural function of endogenous ligands by binding to specific cell-surface receptors.

Disease 52
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Key improvements to Parkinson's disease cell therapies

Science Daily: Pharmacology News

Researchers have demonstrated that a transplant surgical procedure (called 'needle trauma') triggers a profound immune response and causes the death of most grafted dopamine neurons. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.

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Unlocking the power of stem cell therapy

Drug Target Review

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Additionally, ADSCs possess immunomodulatory properties, enabling them to modulate immune responses and promote tissue healing.

Therapies 105
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Could natural terpenes be an alternative for the treatment of neglected tropical diseases?

Chemical Biology and Drug Design

Terpenes are compounds that inhibit the growth of Leishmania spp, Schistosoma spp, and Echinococcus spp, cause changes in parasite ultrastructure, inhibit enzyme activity, and modulate the immune response. Their use in combination therapy decreases the concentration of the reference drug used.

Treatment 100
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Cell and gene therapy development moves into cardiac indications

Drug Target Review

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.