Thursday, July 10, 2014
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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.
Published online July 10, 2014
Patent and licensing status
Fico, A. et al. J.
Neurosci.; Published online July 11, 2014;
Contact: Harold Cremer, Aix-Marseille University, Marseille,
Contact: Rosanna Dono, same affiliation as above
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