Effect of transplantation of mouse marrow stromal cells through injection into cerebrospinal fluid on Cu/ Zn superoxide dismutase 1 transgenic mice

Objective To investigate the effect of transplantation of mouse marrow stromal cells (mMSCs) on Cu/Zn superoxide dismutase 1 (SOD1) transgenic mice through cerebrospinal fluid (CSF) injection. Methods We delivered 5×105m MSCs into SOD1 transgenic mice by single injection to CSF at the age of 8 weeks or by multiple injections at the age of 8，10 and 12 weeks.Neurologic phenotype observation，weight measurement，hanging wire test and motor neuron counts were used to assess disease progression of SOD1 mice. Results Single transplantation of mMSCs didn’t show beneficial effect on SOD1 mice.Compared with vehicle-treated mice，SOD1 transgenic mice with multiple transplantations of m MSCs demonstrated attenuated weight loss，improved motor performance，decreased motor neuron loss，and importantly，prolonged survival by 17 days ((157±15) d vs (140±11) d;χ2=7.56，P<0.01). Conclusions Intrathecal injection could be an effective route of cell transplantation，and multiple transplantations of mMSCs are needed to achieve the therapeutic effect on SOD1 mice. Key words: Amyotrophic lateral sclerosis; Bone marrow transplantation; Cell transplantation; Superoxide dismutase; Mice，transgenic