Allele-specific silencing as therapy for familial amyotrophic lateral sclerosis caused by the p.g376d tardbp mutation

HIGHLIGHTS

• who: Roberta Romano from the (UNIVERSITY) have published the research work: Allele-specific silencing as therapy for familial amyotrophic lateral sclerosis caused by the p.G376D TARDBP mutation, in the Journal: (JOURNAL)
• what: The TDP-43wt full-length construct used in this study has been described previously . In the experiments , the authors used ALS1 and ALS2 cells: which were obtained from affected individuals belonging to the fourth generation of different branches of the family tree. The analysis showed a substantial reduction of GFP-TDP-43 wild-type and GFP-TDP-43G376D transcripts in the presence . . .
  

   

  

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