Licensing status

Publication and contact information

Drug platforms

Glypican 4 (GPC4) deficiency to improve embryonic stem cell (ESC)-derived therapies for Parkinson's disease (PD)

Cell replacement therapies for PD derived from GPC4-deficient ESC lines could offer improved safety and efficacy over cells with intact GPC4. Residual, undifferentiated stem cells in stem cell-derived cell therapies can cause teratoma formation. In culture, mouse ESCs expressing a loss-of-function mutant Gpc4 showed greater differentiation into dopaminergic neurons than wild-type mouse ESCs. In a rat model of PD, injection of the Gpc4-deficient mouse ESCs into the substantia nigra improved motor function and increased survival compared with injection of wild-type mouse ESCs. Next steps could include evaluating long-term outcomes and tumor risk in rodents receiving transplants of neurons derived from the Gpc4-deficient ESCs.

SciBX 7(26); doi:10.1038/scibx.2014.779
Published online July 10, 2014

Patent and licensing status unavailable

Fico, A. et al. J. Neurosci.; Published online July 11, 2014;
Contact: Harold Cremer, Aix-Marseille University, Marseille, France
Contact: Rosanna Dono, same affiliation as above