Antisense and gene therapy options for duchenne muscular dystrophy arising from mutations in the n-terminal hotspot

HIGHLIGHTS

• who: Harry Wilton-Clark and Toshifumi Yokota from the Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta have published the Article: Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot, in the Journal: Genes 2022, 13, 257. of /2022/
• what: The aim of this article is to highlight the current state of development for precision therapies focusing on the currently underserved exon 2-22 hotspot.
• how: These data indicated that vomiting nausea decreased appetite and pyrexia were common adverse events occurring . . .
  

   

  

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