hNT neurons delay onset of motor deficits in a model of amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS) is a degenerative motor neuron disease that manifests as a progressive muscular weakness leading to paralysis and death. Because of the diffuse nature of the motor neuron death, this disease is not considered a good candidate for treatment through neural transplantation. The purpose of this study was to show that transplantation of human neuron-like cells (hNT neurons) into the spinal cord of a transgenic ALS mouse model would improve motor deficits. The hNT neurons were transplanted bilaterally into L4-L5 spinal cord of the transgenic mice ( approximately 8 weeks of age), and the animals were evaluated on health and behavioral measures. The animals were perfused, and immunohistochemistry was performed to identify the transplanted cells. Transplantation of the hNT neurons into the spinal cord delayed the onset of motor behavioral symptoms. This was the first demonstration that even localized transplantation of neural cells directly into the parenchyma could improve motor function in an ALS model. Further study is needed to delineate the mechanism underlying these effects. This therapeutic approach has the potential to restore neural transmission, thereby improving quality of life for the ALS patient and possibly extend life expectancy.
Willing
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AE
Center for Aging and Brain Repair, Department of Neurosurgery, University of South Florida College of Medicine, Tampa, FL 33612, USA. awilling@hsc.usf.edu
Garbuzova-Davis
S
S
Saporta
S
S
Milliken
M
M
Cahill
D W
DW
Sanberg
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PR
D000690:Amyotrophic Lateral Sclerosis / Q000503:physiopathology / Q000601:surgery*; D000818:Animals; D001835:Body Weight / Q000502:physiology; D016380:Brain Tissue Transplantation; D004195:Disease Models, Animal; D006085:Graft Survival / Q000502:physiology*; D008159:Lumbar Vertebrae; D051379:Mice; D008822:Mice, Transgenic; D009069:Movement Disorders / Q000503:physiopathology / Q000601:surgery*; D009474:Neurons / Q000166:cytology / Q000637:transplantation*; D020127:Recovery of Function / Q000502:physiology; D013116:Spinal Cord / Q000601:surgery*; D013482:Superoxide Dismutase / Q000172:deficiency / Q000235:genetics; D000072105:Superoxide Dismutase-1; D016896:Treatment Outcome; D014407:Tumor Cells, Cultured / Q000166:cytology / Q000637:transplantation*
C000606290:SOD1 protein, human; C000606291:Sod1 protein, mouse; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1