This work demonstrates that selection of ASD subjects based on endophenotypes unraveled biologically relevant pathway disruption and revealed a potential cellular mechanism for the therapeutic effect of IGF-1.
Accordingly, IGF-1 should be studied as a potential treatment of ASD and other mental disorders characterized with brain dysconnectivity such as schizophrenia.
We start with reviewing findings on neurotrophic factor levels in human individuals with ASD, continue with providing a broad overview on murine BDNF and IGF-1 in several well-established mouse models of ASD and finally discuss the therapeutic potential of both molecules in the context of translational ASD research.