
bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2025, Номер unknown
Опубликована: Апрель 16, 2025
Summary Establishing long-range connections during human brain development is an intricate multi-step process disturbed in many neurodevelopmental disorders (NDDs). The aberrant formation of these caused by mutations a plethora different genes with distinct molecular functions, triggering the question whether there are common key downstream mediators at which pathologies converging. We employed organoids to model early developmental aspects Coffin-Siris-like 9, Opitz BBB/G, and Pitt-Hopkins syndromes. These NDDs SOX11 , MID1 TCF4 respectively, characterized multitude symptoms yet share alterations projections as feature. Here, we uncover that all three phenotypically converge, showing impaired neurite extension increased tortuosity decreased growth speed resulting shorter beelines. Moreover, mutant neurites exhibit decrease persistence providing conceptual framework explaining why long- but not short-range affected. Correlating converging cellular phenotype, characterization revealed striking convergence on signaling pathways implicated interaction their extracellular environment. In-silico modeling perturbation outgrowth suggest altered neurite-extracellular environment interactions sufficient recapitulate phenotypes also facilitate prediction specific parameters causing neurons.
Язык: Английский