Genetically modified cells were transplanted in a rat middle cerebral artery occlusion model (MCAO).
However, the researchers were able to correct phenotypic weight loss if they first expanded these modified cells ex vivo before transplanting them back into the mice.
In future experiments, he hopes to transplant only modified cells.
A third category of research, using stem cells or genetically modified cells (e.g., glial cells) that are transplanted into the zone of injury, seeks to replace damaged cells with substitute neurons or other cells that can restore function.
However, safety concerns about in vivo delivery of viral vectors and poor post-transplant survival of ex vivo modified cells remain key hurdles for clinical translation of gene therapy.
Therefore, based on these and other published reports/reviews about mesenchymal stromal cells [ 38] and supported by our findings, we conclude that new techniques need to be developed in order to safely culture and genetically modify hMSC cell transplants for research, and eventually clinical applications.
Clinical cell transplant trials have now been performed in Parkinson's disease, Huntington's disease, demyelinating diseases, retinal disorders, stroke, epilepsy, and even deafness, and normally are designed as cell replacement strategies, although implantation of genetically modified cells for supplementation of growth factors has also been tried.
The 'gene-corrected' cells are then re-administered to the patient in the form of an 'autologous gene modified CD34 + cell' transplant.
The stem cell transplants replace normal cells killed in chemotherapy.
Other uses of stem cells could provide income before cell transplants become feasible.
