Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expresse
Hemophilia mice were treated for one month with ASOs targeting either Upf1 or Upf3b. The liver RNAs were extracted and analyzed Overall design: Hemophilia mice were treated for one month with ASOs tar
