Neuropathology of phenylacetate poisoning in rats: an experimental model of phenylketonuria.

Results of this investigation indicate that the suckling rat treated with phenylacetate should be a useful new model for studying the pathogenesis of phenylketonuria and neuronal development. Both cerebellar and retinal neurons of postnatally treated rats are vulnerable to the adverse effects of phenylacetate. Morphological changes observed in the cerebellum, retina, and optic nerve of treated animals during the fourth to twenty-first days of life consist of regional reduction in the size of cerebellar vermis lobules IV, V, VIa, and IX, 35 to 40% reduction in thickness of the molecular layer, accumulation of cerebellar external granular cells and retinal neuroblastic cells, fewer parallel fibers in the cerebellar cortex, and fewer myelinated axons in the optic nerve.