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AAN: Stem Cells Show Promise in Mouse Model of ALS

Intrathecal or intravenous injection of neural stem cells prolongs disease duration and lifespan

AAN: Stem Cells Show Promise in Mouse Model of ALS

THURSDAY, Jan. 10 (HealthDay News) -- Implantation of a neural stem cell (NCS) fraction of induced pluripotent stem cells (iPSCs) shows promise in a mouse model of amyotrophic lateral sclerosis (ALS), according to a study released in advance of its presentation at the annual meeting of the American Academy of Neurology, which will be held from March 16 to 23 in San Diego.

Stefania Corti, M.D., Ph.D., from the University of Milan, and colleagues used a non-viral, non-integrating method based on the expression of reprogramming factors with episomal vectors to generate iPS cell lines from human skin fibroblasts. A protocol to promote NSC fate was used to differentiate iPSCs. Based on their high ALDH activity and low side scatter (ADLHhiSSClo), a primitive NSC fraction was isolated. iPSC-purified NSCs were transplanted either intrathecally or by systemic intravenous injection into SOD1G93A mice (mouse model of ALS).

The researchers found that ALDH(hi)SSC(lo) NSCs from iPSCs are self-renewing and multipotent. In vitro, they were able to differentiate into the three neuroectodermal lineages. NSCs (both intrathecally and systemically grafted) migrated into the parenchyma and engrafted the host spinal cord, where they expressed neuronal precursors- and neuronal mature-specific markers. In the mouse model of ALS, cell transplantation prolonged disease duration and lifespan, promoted survival of motor neurons, and improved neuromuscular function.

"ALS is a fatal, progressive, degenerative disease that currently has no cure. Stem cell transplants may represent a promising avenue for effective cell-based treatment for ALS and other neurodegenerative diseases," Corti said in a statement.

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