Stem Cells Engineered to Express GDNF and VEGF Improve Survival in ALS Rats

In a study published in Molecular Therapy on May 28, researchers led by Dr. Masatoshi Suzuki, assistant professor of comparative biosciences at the University of Wisconsin-Madison School of Veterinary Medicine, showed that treating SOD1 rats with modified human mesenchymal stem cells (hMSC) delayed disease onset and prolonged survival in the rats. The modified hMSC were engineered to express one of four growth factors, including brain-derived neurotrophic factor (BDNF), vascular endothelial growth factor (VEGF), insulin-like growth factor-I (IGF-I), or glial cell line-derived neurotrophic factor (GDNF). Intriguingly, the four factors did not have the same effects; hMSC’s expressing IGF-I or BDNF had no observable effect, while treatment with hMSC’s expressing VEGF or GDNF “prolonged survival and slowed the loss of motor function.” However, the researchers unexpectedly observed the greatest effects when they treated the rats with both VEGF and GDNF.


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