Sara Abdollahi - Department of Pathobiology, School of Veterinary Medicine, Shiraz University, Shiraz, Iran|Shefa Neuroscience Research Center, Khatam Alanbia Hospital, Tehran, Iran
Azizollah Khodakaram-Tafti - Department of Pathobiology, School of Veterinary Medicine, Shiraz University, Shiraz, Iran
Hadi Aligholi - Department of Neuroscience, School of Advanced Medical Sciences and Technologies, Shiraz University of Medical Sciences, Shiraz, Iran
Saeid Ziaei - Department of Basic Sciences, Faculty of Paramedical Sciences, Shahid Beheshti University of Medical Sciences, Tehran, Iran

Objective(s): Neural stem/progenitor cells (NS/PCs) hold a great potential for delivery of therapeutic agents into the injured regions of the brain. Efficient gene delivery using NS/PCs may correct a genetic defect, produce therapeutic proteins or neurotransmitters, and modulate enzyme activation. Here, we investigated the efficiency of a recombinant lentivirus vector expressing green fluorescent protein (GFP) for genetic engineering of human NS/PCs obtained during brain surgery on patients with medically intractable epilepsy.Materials and Methods: NS/PCs were isolated from human epileptic neocortical tissues. Three plasmids (pCDH, psPAX2, pMD2.G) were used to make the virus. To produce the recombinant viruses, vectors were transmitted simultaneously into HEk-293T cells. The lentiviral particles were then used to transduce human NS/PCs. Results: Our in vitro study revealed that lentivirus vector expressing GFP efficiently transduced about 80% of human NS/PCs. The expression of GFP was assessed as early as 3 days following exposure and remained persistent for at least 4 weeks. Conclusion: Lentiviral vectors can mediate stable, long-term expression of GFP in human NS/PCs obtained from epileptic neocortical tissues. This suggests lentiviral vectors as a potential useful tool in human NS/PCs-based gene therapy for neurological disorders, such as epilepsy.

GFP, Lentivirus, Neural stem/progenitor cells, Seizure, Transplantation