Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy

A simple clinical neurological test was developed to evaluate response to gene therapy in a preclinical canine model of X-linked myotubular myopathy (XLMTM). This devastating congenital myopathy is caused by mutation in the myotubularin (MTM1) gene. Clinical signs include muscle weakness, early resp...

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Bibliographische Detailangaben
Veröffentlicht in:Hum Gene Ther Clin Dev
Hauptverfasser: Snyder, Jessica M., Meisner, Allison, Mack, David, Goddard, Melissa, Coulter, Ian T., Grange, Robert, Childers, Martin K.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2015
Schlagworte:
Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4574743/
https://ncbi.nlm.nih.gov/pubmed/26086764
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.049
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