Article
Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.
PloS one - 1 Jan 2016
Sintusek Palittiya, Catapano Francesco, Angkathunkayul Napat, Marrosu Elena, Parson Simon H, Morgan Jennifer E, Muntoni Francesco, Zhou Haiyan
Abstract excerpt
Gastrointestinal (GI) defects, including gastroesophageal reflux, constipation and delayed gastric emptying, are common in patients with spinal muscular atrophy (SMA). Similar GI dysmotility has been identified in mouse models with survival of motor neuron (SMN) protein deficiency. We previously described vascular defects in skeletal muscle and spinal cord of SMA mice and we hypothesized that similar defects...
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