New research points to innovative approaches in neuroscience and rare disorders. AAV-OTOF gene therapy shows promise restoring hearing in children and adults. Studies reveal microglia replacement halts progression in rare ALSP brain disease in both mice and patients. Four distinct autism subtypes linked to genetics were identified, suggesting pathways to personalized care. Additionally, novel materials from sea cucumbers may lead to potent cancer enzyme inhibitors, and gut microbiome metabolites have been implicated in vascular aging, highlighting complex host-microbe interactions relevant to age-related diseases.