The motor neuron disease spinal muscular atrophy (SMA) is the second most common genetic disorder leading to death in childhood. There is currently no cure for SMA, but some clinicians and researchers consider stem cell transplantation as a potential therapeutic strategy. And now, Giacomo Comi and colleagues, at the University of Milan, Italy, have generated data using a mouse model of SMA to suggest that spinal cord neural stem cells (NSCs) might be a possible treatment for individuals with SMA.
In the study, NSCs from mice in which a green marker protein was expressed only in nerve cells known as motor neurons (the cells that are defective in SMA) were transplanted into the fluid bathing the spinal cord of mice with an SMA-like disease. The transplanted cells developed into a small number of motor neurons and the treated mice showed improved muscular function and increased lifespan, when compared with untreated mice. Further analysis indicated that the major effect of NSC transplantation was that the transplanted cells improved the survival and function of the motor neurons already in the mice, making them more like normal motor neurons (at the gene expression level). The authors therefore suggest that in the future, NSCs might be used in the development of therapeutic protocols for the treatment of SMA and other motor neuron diseases.
TITLE: Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy
AUTHOR CONTACT:
Giacomo P. Comi
University of Milan, Milan, Italy.
Phone: 390255033817; Fax: 390250320430; E-mail: giacomo.comi@unimi.it.
View the PDF of this article at: https://www.the-jci.org/article.php?id=35432
Journal
Journal of Clinical Investigation