AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion

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In: Science & Nature

Main Adeno-associated virus (AAV) vectors proved to be effective and safe for hepatocyte-targeted reversal of factor IX and VIII deficiency in hemophilia B and A, respectively, with the AAV5 capsid being the first to receive Food and Drug Administration (FDA) approval1,2. However, these achievements of therapeutic efficacy cannot be readilyContinue Reading