Correction of clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy

HIGHLIGHTS

• who: Ningyan Hu from the Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA have published the article: Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy, in the Journal: (JOURNAL) of 21/03/2023
• what: By contrast, the splice-shifting morpholino chemistry that the authors have used in this study has demonstrated safety in humans, with four drugs already FDA-approved for the treatment of Duchenne muscular dystrophy43.
• how: The authors used a double homozygous model of DM1 and antisense oligonucleotide exon . . .
  

   

  

  If you want to have access to all the content you need to log in!

  Thanks :)

  Username or Email

  Password

  Remember me

  Lost your password?

  If you don't have an account, you can create one here.