clinical trials is done almost entirely through notoriously imprecise clinical diagnostic criteria. Gene network and neurobiological analyses on hiPSC-derived neurodevelopmental models could be used instead to identify subtypes based on specific genes/pathways that are altered. Finally, another therapeutic avenue may be through transplantation. While this is already in clinical trial for some adult neurological disorders such as Parkinson’s disorder, we believe it is challenging for disorders of childhood, especially for neurodevelopmental disorders where, as demonstrated by this review, there isn’t much evidence of neuronal loss or neural tissue damage that is in need of being regenerated. One recent clinical trial, not directly regarding neurodevelopmental disorders but in the area of CNS disorders, is the use of hiPSC-derived retinal pigment epithelial cells for the treatment of macular degeneration 181. Broadly, the idea of editing mutations in patient-derived hiPSCs and then transplanting them back into the patient is exciting. This is mainly because transplantation normally is accompanied by host rejection risk, however this would not the case with hiPSCs as the cells are derived from the patient themselves.
