PQBP1 is a protein functioning in maturation of RNA. Especially, PQBP1 is important for RNAs whose functions are related to neurons in the brain or to neural stem cells in the embryonic brain. If the function of PQBP1 is disturbed in children’s or adult brains, brain neurons cannot have sufficient contacts among them via the connection machineries called as synapse. Also if the function is disturbed in neural stem cells of embryo brains, patient brains cannot become large enough and become microcephaly. Recently, PQBP1 is also known to function in Alzheimer’s disease. One case is the deficiency of PQBP1 due to amyloid peptide accumulation in neurons, and another case is the inflammation in the brain by PQBP1-mediated and Tau-induced activation of immune cells in the brain. In addition, PQBP1 is necessary for immune defence against human immunodeficiency virus (HIV) contrarily.