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Drug design, drug delivery and technologies
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Illustration of brain receptors with their neurotransmitters
Neurology/psychiatric

New GluN1/GluN3A NMDA receptor inhibitors based on AI

June 6, 2025
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Scientists at Shanghai Tech University have used the scaffold-hopping artificial intelligence model Geminimol to identify N-methyl-D-aspartate (NMDA) receptor ligands that show selectivity and specificity. The researchers found that GM-10 could be a potent inhibitor of the GluN1/GluN3A subunits of the NMDA receptor, demonstrating the utility of this technique to develop new drugs.


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Illustration of mast cell emitting granules
Drug design, drug delivery & technologies

Dual-action synthetic peptides could fight superbugs

June 2, 2025
By Mar de Miguel
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A peptide with a dual mechanism of action – it dissolves the bacterial membrane and activates the immune system – could be an effective weapon against microorganisms that have evolved ways to evade antibiotics, as superbugs do. Scientists at the University of Pennsylvania (UPenn) have designed stable synthetic peptides that activate mast cell receptors, which are cells involved in the innate and adaptive immune response. This dual approach eliminates bacteria and recruits neutrophils to finish the job.
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Art concept for mouse model
Drug design, drug delivery & technologies

From mice to big animal models in gene therapy for rare diseases

May 23, 2025
By Mar de Miguel
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The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
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Silhouette of head and brain with DNA double helixes
Neurology/psychiatric

Armamentarium, the new genetic weapon to study brain disorders

May 22, 2025
By Mar de Miguel
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A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.
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DNA double helix under a magnifying glass
Drug design, drug delivery & technologies

Base and prime editions to achieve genetic cure

May 21, 2025
By Mar de Miguel
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Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
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