Project Co-Funded with a Grant from Families of Spinal Muscular Atrophy and SMA Europe Shows Embryonic Stem Cell-Derived Neural Cells Improve Spinal Muscular Atrophy in Mice.
January 4, 2010.

This study was recently published in the journal Brain by lead author Dr. Stefania Corti in the laboratory of Dr. Giacomo Comi at the Centre of Excellence on Neurodegenerative Diseases at the University of Milan.  

The authors have previously published that primary neural stem cells derived from spinal cord of mice can ameliorate the spinal muscular atrophy phenotype in mice.  However,  this primary source has limited value in therapy development.  Therefore, the authors have gone on to illustrate that neural cells derived from pluripotent embryonic stem cells show the same potential therapeutic benefit as those derived from spinal cord, offering promise as an unlimited source of neural stem cells for therapeutic transplantation and intervention.

The authors demonstrated in the current study that embryonic stem cell-derived neural stem cells can differentiate into motor neurons. In addition, following their intrathecal transplantation into spinal muscular atrophy mice, the neural stem cells survived and migrated to appropriate areas.  Transplantation resulted in improved motor function, enhanced lifespan, and exhibited neuroprotective capability in the mice.  
According to the authors, these results suggest translational potential for the use of pluripotent cells in neural stem cell-mediated therapies.

Please click here to read the abstract of this article.

The full paper reference is:
Embryonic stem cell-derived neural stem cells improve spinal muscular atrophy phenotype in mice.
Corti S, Nizzardo M, Nardini M, Donadoni C, Salani S, Ronchi D, Simone C, Falcone M, Papadimitriou D, Locatelli F, Mezzina N, Gianni F, Bresolin N, Comi GP.
Brain. 2009 Dec 23.