Article

Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

2021-09-21

Abstract excerpt

<title>Abstract</title> <p>Liver gene therapy with adeno-associated viral (AAV) vectors delivering a clotting factor transgene into hepatocytes has shown multi-year therapeutic benefit in adults with hemophilia. However, anti-AAV pre-existing immunity and the mostly episomal nature of AAV vectors, currently challenges application of AAV-vector mediated liver gene therapy to people with anti-AAV neutralizing antib...

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Literature Corpus work: df13d810-b59d-5c9d-a4fd-cd0b7ece3c52
DOI: 10.21203/rs.3.rs-858195/v1

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