Abstract
Neurturin (NTN) belongs to the glial cell line-derived neurotrophic factor (GDNF) family of ligands (GFL) and has been found to be a potent survival factor for midbrain dopaminergic neurons. Therefore, it is an attractive candidate protein for the gene therapy of Parkinson's disease. Like other GFLs, mature NTN is secreted from producer cells upon cleavage of a precursor protein at an RXXR site recognized by furin-like convertases. We have produced adeno-associated virus type 2 (AAV2)-based vectors encoding human NTN (hNTN) under the control of the CAG promoter and observed that upon transduction of cells in vitro or in vivo, hNTN expression remained essentially cell bound. Only low levels of secreted protein could be detected in the extra-cellular compartment in rat brain. In tissue culture supernatants, secreted protein was not detected. In contrast to these observations, rodent NTN has been shown to be efficiently secreted from cells.
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