Long-term efficacy of aav9-u7snrna-mediated exon 51 skipping in mdx52 mice

HIGHLIGHTS

• who: Philippine Aupy from the Saclay Institute de Recherche en Myologie, Paris, France have published the research work: Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice, in the Journal: (JOURNAL) of 17/06/2020
• what: The authors examined the therapeutic potential of an AAV-U7snRNA targeting the human DMD exon 51 which could be applicable to 13% of DMD patients. The authors evaluated the ability of an AAV9 (AAV serotype 9)-U7 exon 51 (U7ex51) vector to induce exon 51 skipping and to restore expression of the full-length dystrophin . . .
  

   

  

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