Antisense morpholino-based in vitro correction of a pseudoexon-generating variant in the sgcb gene

HIGHLIGHTS

• who: Francesca Magri and colleagues from the Neurology Unit, IRCCS Fondazione Ca` Granda Ospedale Maggiore Policlinico, Milan, Italy Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan have published the Article: Antisense Morpholino-Based In Vitro Correction of a Pseudoexon-Generating Variant in the SGCB Gene, in the Journal: (JOURNAL)
• what: The authors report a novel LGMDR4 patient presenting as almost asymptomatic at 12 years of age and with repeated findings of high creatine-kinase levels. The establishment of patient-specific induced pluripotent stem_cells provided the work bench to test a morpholino oligomer . . .
  

   

  

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