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Updated: Jul 12, 2026
Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
Published on: February 24, 2026
Human mesenchymal stromal cells ameliorate the phenotype of SOD1-G93A ALS mice
1Department of Neurology, First Affiliated Hospital, Sun Yat-sen University, Guangzhou, PR China.
Background:
Amyotrophic lateral sclerosis (ALS) is a progressive, lethal, neurodegenerative disease, currently without any effective therapy. Multiple advantages make mesenchymal stromal cells (MSC) a good candidate for cellular therapy in many intractable diseases such as stroke and brain injury. Until now, no irrefutable evidence exists regarding the outcome of MSC transplantation in the mouse model of ALS. The present study was designed to investigate the therapeutic potential of human MSC (hMSC) in the mouse model of ALS (SOD1-G93A mice).
Methods:
hMSC were isolated from iliac crest aspirates from healthy donors and kept in cell cultures. hMSC of the fifth passage were delivered intravenously into irradiated pre-symptomatic SOD1-G93A mice. Therapeutic effects were analyzed by survival analysis, rotarod test, motor neuron count in spinal cord and electrophysiology. The engraftment and in vivo differentiation of hMSC were examined in the brain and spinal cord of hMSC-transplanted mice.
Results:
After intravenous injection into irradiated pre-symptomatic SOD1-G93A mice, hMSC survived more than 20 weeks in recipient mice, migrated into the parenchyma of brain and spinal cord and showed neuroglia differentiation. Moreover, hMSC-transplanted mice showed significantly delayed disease onset (14 days), increased lifespan (18 days) and delayed disease progression compared with untreated mice.
Discussion:
Our data document the positive effects of hMSC transplantation in the mouse model of ALS. It may signify the potential use of hMSC in treatment of ALS.
