A limb-girdle muscular dystrophy 2I model of muscular dystrophy identifies corrective drug compounds for dystroglycanopathies.
Serafini PR, Feyder MJ, Hightower RM, Garcia-Perez D, Vieira NM, Lek A, Gibbs DE, Moukha-Chafiq O, Augelli-Szafran CE, Kawahara G, Widrick JJ, Kunkel LM, Alexander MS.
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JCI Insight. 2018 Sep 20;3(18):e120493. doi: 10.1172/jci.insight.120493. eCollection 2018 Sep 20.
JCI Insight. 2018.
PMID: 30232282
Free PMC article.