Early maldevelopment of brain tissue has been postulated as a crucial factor in the pathogenesis of schizophrenia. The neurotrophin brain-derived neurotrophic factor (BDNF) is widely and abundantly expressed in mammalian brain. BDNF plays important role in the development of brain and has critical effects on the differentiation of monoaminergic neurons. In rodents, antipsychotic treatments have been shown to alter the expression of brain BDNF. Furthermore, schizophrenia is associated with a decrease in central as well as peripheral BDNF protein concentration. Thus, in light of this evidence, the BDNF gene is considered as an attractive candidate gene for predicting schizophrenia or antipsychotic therapeutic response. In this review, we summarize the investigations of BDNF levels and the BDNF genetic effects on therapeutic response and the risk for schizophrenia, proposing several recommendations for future genetic studies of BDNF signaling pathways in schizophrenia.