Engineering Approach
Target Overexpression-Lentivirus
Alternative Names
ATF; ATF1; ATF2; HSCR3; HFB1-GDNF
Full Name
Glial cell derived neurotrophic factor
Introduction
This gene encodes a secreted ligand of the TGF-beta (transforming growth factor-beta) superfamily of proteins. Ligands of this family bind various TGF-beta receptors leading to recruitment and activation of SMAD family transcription factors that regulate gene expression. The encoded preproprotein is proteolytically processed to generate each subunit of the disulfide-linked homodimer. The recombinant form of this protein, a highly conserved neurotrophic factor, was shown to promote the survival and differentiation of dopaminergic neurons in culture, and was able to prevent apoptosis of motor neurons induced by axotomy. This protein is a ligand for the product of the RET (rearranged during transfection) protooncogene. Mutations in this gene may be associated with Hirschsprung disease and Tourette syndrome. This gene encodes multiple protein isoforms that may undergo similar proteolytic processing.
Applications
Provide significant neuronal protection in Parkinson's disease
Reference
Zhao, Yuling et al. "GDNF-transfected macrophages produce potent neuroprotective effects in Parkinson's disease mouse model." PloS one vol. 9,9 e106867. 17 Sep. 2014, doi:10.1371/journal.pone.0106867