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Multi-region brain organoids for unprecedented insights into human neurodevelopmental disorders
08 July 2025 Despite extensive biomedical research, disease-modifying treatments for human neurodevelopmental disorders such as autism spectrum disorder and schizophrenia are still lacking. Neurology and psychiatry are consistently the lowest success categories in drug development, with a 95-97% failure rate in clinical trials. Owing to inter-species differences in brain anatomy and physiology, animal models do not recapitulate human-specific molecular and cellular pathways


Genetic Engineering Unlocks Organotypic Vascularization
02 October 2025 Microvessels - capillaries and post‑capillary venules - are the smallest vessels in the circulation, with a cellular composition and thin-walled structure that enables nutrient exchange, oxygen delivery, waste removal, and immune‑cell trafficking between the blood and surrounding tissue cells. The absence of microvascular function is often cited as a major bottleneck of in vitro preclinical models, leading to tissue necrosis and limiting physiological releva


Muotri Lab's Pitt Hopkins syndrome patient-derived brain organoids lay the foundation for gene therapy trial
31 January 2026 Mahzi Therapeutics-sponsored Phase 1/2 study to assess safety and efficacy of MZ-1866 gene therapy for children and adults with Pitt Hopkins syndrome is about to start. The trial was announced by Mahzi in 2025, highlighting the sponsor's collaboration with the Pitt Hopkins Research Foundation and the Muotri Lab at UC San Diego in developing this long awaited treatment. Pitt-Hopkins syndrome is a rare genetic disorder caused by mutations in the transcript
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