Review Article

Translational Research in Stem Cell Treatment of Neuromuscular Diseases

Table 3

Table summarizing the preclinical studies on the treatment of MS with stem cells.

Stem cellAnimal modelResultReferences

Mouse BM-derived HSCsNeonatal mouseInjected stem cells differentiated into oligodendroglial cells in vivo[22]
Rat NPCsRatTransplanted cells had migrated into white matter and acquired specific markers of the astroglial and oligodendroglial lineages[82]
Mouse NPCsMouseSignificant numbers of donor cells entered into demyelinating areas of the central nervous system and differentiated into mature brain cells[83]
Mouse NPCs
MouseThe size of demyelinated areas and, extent of chronic axonal pathology were reduced in the transplanted brains[84]
Mouse NPCs
MouseDecreased CNS inflammation and tissue injury and attenuated severity of EAE[85]
Mouse NPCs
MouseTransplanted NPCs accumulated in inflammatory demyelinating lesions[86]
Mouse NPCsMouseDecreased inflammation in brain but no effect in spinal cord lesions[87]
Mouse NPCsMouseReduced migration of transplanted cells in chronic disease[88]
Mouse NPCsMouseEnhanced remyelination[84]
Mouse NPCsMouseNPC differentiated into oligodendrocytes in vivo in long term[87]
Mouse NPCs committed to glial lineageMouseMigration of cells from the implantation site and remyelination of axons. Behavioral improvement[89]
Mouse NPCs committed to glial lineageMouseRemyelination of the axons was observed without alteration of T cells or macrophages within the CNS [90]
Human NPCsMonkeyDecreased disability and increased survival as well as long-term survival of implanted cells[23]
Human ESC-derived NPCsMouseClinical improvement, cells migrated to the host white matter, attenuation of the inflammatory process[91]
Monkey NPCsMonkeySignificant number of myelinating profiles in the transplantation zone[4]
BM transplantationMouseComplete amelioration of the symptoms of MS[92]
BM transplantationMouseBM transplantation effectively blocked or delayed EAE development[93]
BM transplantationMouseProtection from disease development[94]
NT2 embryonal carcinoma cell line,
BM transplantation
MouseBoth types of cells remained viable in the mouse brain and differentiated into neurons, astrocytes, and oligodendrocytes[95]
Mouse NSCsMouseNSCs migrated and differentiated into oligodendrocytes and stimulated remyelination[96]
Mouse NSCs and mouse BMC-derived NSCsMouseBoth cell types suppressed the inflammatory process[97]
Human BMCsMouseDemyelination and the number of the lesions have decreased[98]
Mouse NSCsMouseLimited or no therapeutic potential [25]
Mouse BMCsMouseAntioxidant and neuroprotective activity for MSCs was documented[99]
Mouse BMCsMouseDecreased inflammation and decreased tissue levels of inflammatory mediators[100]
Mouse BMCsMouseBMCs enhanced recovery, prevented relapses, and promoted myelin repair[101]
Mouse BMCsMouseTransplanted cells decreased the inflammation but also formed demyelinating lesions in the brain parenchyma in some cases[102]
Human BMCsMouseInjected BMCs accumulated in the CNS, reduced the extent of damage, and increased oligodendrocyte lineage cells in lesion areas[103]
Human BMCs differentiated into neurotrophic factor-producing cellsMouseDelay of symptom onset and increased animal survival[104]
TREM2*-transduced
BM-derived myeloid precursor cells
MouseLimited tissue destruction and facilitated repair[105]
IFN- ** gene transfected mouse BMCsMouseInhibition of disease onset and decrease in clinical severity.[106]
BDNF*** gene transfected mouse BMCs MouseDelayed disease onset and a reduction in overall clinical severity[107]
Mouse ASCsMouseBimodal therapeutic potential of ASCs was documented via suppressing the autoimmune response and via inducing local neuroregeneration [108]
SC and SCPs RatSCPs survived well, migrate through normal CNS tissue, interface smoothly and intimately with host glial cells, and myelinate extensively among the astrocytes of the retina[109]
Mouse OPCs and BMCsMouseBMCs increased OPC engraftment, migration, and maturation in myelinating oligodendrocytes[110]
Mouse iPS derived OPCMouseiPS cells could be differentiated into functional OPCs[24]
OPC line CG-4RatTransplanted cells migrated to inflammatory lesions and differentiated into oligodendrocytes[111]

*Triggering receptor expressed on myeloid cells 2, **interferon- , ***brain-derived neurotrophic factor.