Glial cell line-derived neurotrophic factor

Glial cell line-derived neurotrophic factor (GDNF) is a protein that, in humans, is encoded by the GDNF gene. GDNF is a small protein that potently promotes the survival of many types of neurons.

Function

GDNF is a glycosylated, disulfide-bonded homodimer that is a member of the transforming growth factor β superfamily of growth factors. The protein is expressed in the central nervous system (CNS), peripheral nervous system (PNS), and other tissues such as the kidney and skeletal muscle. GDNF signals through a multicomponent system of receptors that includes a common signal-transducing component (the RET proto-oncogene) and a GDNF family receptor α (GFRα) that confers ligand specificity.

Clinical significance

GDNF has been shown to promote the survival and differentiation of dopaminergic neurons in culture. Mutations in this gene may be associated with Hirschsprung disease. The role of GDNF in the development and maintenance of neurons has led to research into its potential therapeutic uses, particularly in Parkinson's disease.

Research

Research into the therapeutic potential of GDNF has been ongoing since its discovery. Preclinical studies have shown that GDNF can protect and restore damaged dopaminergic neurons in animal models of Parkinson's disease. Clinical trials in humans, however, have produced mixed results.

See also

• Neurotrophic factor
• Neuron
• Parkinson's disease