Genetic Manipulation in Organoid Models and Their Applications in Central Nervous System Pathologies

Recently developed organoid technology represents a novel method of accurately mimicking in vivo conditions by simulating tissue architecture and intercellular interactions in 3 dimensions. Three-dimensional (3D) organoids can provide a more precise representation of in vivo drug action, as well as gene expression and cell differentiation, making them a valuable tool for testing newly developed drugs and simulating various pathologies of the central nervous system. By genetically modifying the cellular component of these organoids, one can induce differentiation into various cells of the human body, ranging from neurons to cells of the gastrointestinal tract, enabling the creation of 3D models of diverse organs of the human body. Here, we discuss various methods that can be used to induce genetic change in cells found in 3D media and possible applications of genetically modified organoids, such as their use in the simulation of pathologies of the central nervous system, and potential therapeutic application of optogenetics within these models. Clinical trial number: not applicable.