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 research: 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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