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Updated: May 5, 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
[Effect and mechanism of marrow stromal cells within cerebrospinal fluid on SOD1 transgenic mice]
Chang Zhou1, Chen Zhang, Qian-qian Yao
1Department of Neurology, Affiliated Hospital of Medical College, Qingdao University, Qingdao 266003, China.
Objective:
To explore the effect and mechanism of murine marrow stromal cells (MSCs) within cerebrospinal fluid (CSF) on Cu/Zn superoxide dismutase 1 (SOD1) transgenic mice.
Methods:
A dose of 5×10(5) MSCs was injected into the CSF of SOD1 transgenic mice at the ages of 8, 10 and 12 weeks.Hanging wire test and motor neuron counts were used to assess disease progression of SOD1 mice. To examine the glial activation in murine lumbar spinal cord at 15 weeks of age, the sections were stained with antibody to CD11b and GFAP through immunohistochemistry.In vitro, mRNA expression of neurotrophin of MSCs was analyzed by reverse transcription-polymerase chain reaction (RT-PCR).
Results:
Compared with vehicle-treated mice, intrathecally transplanted MSCs enhanced motor performance and decreased motor neuron loss. An intrathecal injection of MSCs inhibited microglial (52 ± 7 vs 38 ± 7; F = 69.3, P = 0.02) and astrocyte (79 ± 9 vs 63 ± 9; F = 40.7, P = 0.03) activity in lumbar spinal cord at the age of 15 weeks of age. MSCs cultured in an inflammatory environment expressed a higher mRNA level of neurotrophin (P < 0.05).
Conclusion:
A CSF administration of MSCs has therapeutic effects on SOD1 mice. And the meditation of MSCs may be achieved through inhibiting neuroinflammation and secreting a variety of trophic factors.
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