Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by deficiency of the survival of motor neuron (SMN) protein, which leads to synaptic defects and spinal motor neuron death. Neuromuscular junction (NMJ) abnormalities have been found to be involved in SMA pathogenesi...

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Veröffentlicht in:	PloS one 2016-04, Vol.11 (4), p.e0154723-e0154723
Hauptverfasser:	Lin, Te-Lin, Chen, Tai-Heng, Hsu, Ya-Yun, Cheng, Yu-Hua, Juang, Bi-Tzen, Jong, Yuh-Jyh
Format:	Artikel
Sprache:	eng
Schlagworte:	Abnormalities Amyotrophic lateral sclerosis Animals Antibodies Antisense oligonucleotides Atrophy Biology and Life Sciences Bungarotoxin Care and treatment Cell death Cell survival Denervation Disease Models, Animal Genetic aspects Medical screening Medicine and Health Sciences Mice Mice, Knockout Morpholinos - therapeutic use Motor neuron diseases Motor Neurons - pathology Muscle, Skeletal - innervation Muscles Muscular Atrophy, Spinal - genetics Muscular Atrophy, Spinal - pathology Muscular Atrophy, Spinal - therapy Nerve Degeneration - genetics Nerve Degeneration - pathology Nerve Degeneration - therapy Nerve endings Neuromuscular diseases Neuromuscular Junction - genetics Neuromuscular Junction - pathology Neuromuscular junctions Oligonucleotides Oligonucleotides, Antisense - therapeutic use Pathogenesis Physiological aspects Receptors Research and Analysis Methods Rodents Science SMN protein Spinal muscular atrophy Studies Survival Survival of Motor Neuron 1 Protein - genetics Synaptophysin Transport buildings, stations and terminals
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