
(Folate receptor alpha), a GPI-anchored protein is critical for embryonic development. Disruption of both FRα alleles in mice results in pups with a range of malformations and is lethal to the embryos at the time of neural tube closure. Recent body of evidences emphasizes its role in neural tube defects, cerebral folate deficiency, autism and autism spectrum disorders. Circulating autoantibodies against FRα and cerebral folate deficiency appear to play a crucial role in the cause and pathogenesis of a particular subgroup of autism spectrum disorders with co-existing neurological deficits. Since FRα is known to be over-expressed in cancer cells, it has found a novel theranostic role in cancer diagnosis and treatment by using FA-conjugated imaging agents as diagnostic tools and FA-conjugated nanotherapeutics and immunotherapy for cancer. This review highlights some recent advances and novel roles of FRα other than it being just a folate transporter.
Keywords: Folate receptor alpha, cerebral folate deficiency, autism spectrum disorder, cancer, folate conjugates