Cellular therapies in motor neuron diseases.
Amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) are prototypical motor neuron diseases that result in progressive weakness as a result of motor neuron dysfunction and death. Though much work has been done in both diseases to identify the cellular mechanisms of motor neuron dysfunction, once motor neurons have died, one of potential therapies to restore function would be through the use of cellular transplantation. In this review, we discuss potential strategies whereby cellular therapies, including the use of stem cells, neural progenitors and cells engineered to secrete trophic factors, may be used in motor neuron diseases. We review pre-clinical data in rodents with each of these approaches and discuss advances and regulatory issues regarding the use of cellular therapies in human motor neuron diseases.
Nayak
Mamatha S
MS
Department of Neurology, Johns Hopkins University School of Medicine, 600 North Wolfe Street, Baltimore, MD 21287, USA.
Kim
Yun-Sook
YS
Goldman
Michelle
M
Keirstead
Hans S
HS
Kerr
Douglas A
DA
D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000628:therapy*; D000818:Animals; D030801:Animals, Genetically Modified; D002454:Cell Differentiation; D006801:Humans; D036341:Intercellular Signaling Peptides and Proteins / Q000493:pharmacokinetics; D051379:Mice; D008959:Models, Neurological; D016472:Motor Neuron Disease / Q000628:therapy*; D009046:Motor Neurons / Q000502:physiology*; D051381:Rats; D033581:Stem Cell Transplantation / Q000331:legislation & jurisprudence / Q000592:standards*; D013234:Stem Cells / Q000502:physiology*
D036341:Intercellular Signaling Peptides and Proteins