In hemophilia B gene therapy, either wild-type or Padua factor IX with a liver-specific promoter is inserted into the genome of the AAV vector and administered via a peripheral vein. The vector transduces hepatocytes, leading to the production of FIX. The levels of FIX in patients and unaffected individuals before and after gene therapy are shown below. Professional illustration by Somersault18:24.

In hemophilia B gene therapy, either wild-type or Padua factor IX with a liver-specific promoter is inserted into the genome of the AAV vector and administered via a peripheral vein. The vector transduces hepatocytes, leading to the production of FIX. The levels of FIX in patients and unaffected individuals before and after gene therapy are shown below. Professional illustration by Somersault18:24.

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