Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A
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DOI: 10.15252/emmm.202115199
Abstract: Liver gene therapy with adeno‐associated viral (AAV) vectors is under clinical investigation for haemophilia A (HemA), the most common inherited X‐linked bleeding disorder. Major limitations are the large size of the F8 transgene, which makes… read more here.
Keywords: trans splicing; intein mediated; liver gene; gene therapy ... See more keywords