Article
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
The New England journal of medicine - 18 Nov 2021
George Lindsey A, Monahan Paul E, Eyster M Elaine, Sullivan Spencer K, Ragni Margaret V, Croteau Stacy E, Rasko John E J, Recht Michael, Samelson-Jones Benjamin J, MacDougall Amy, Jaworski Kristen, Noble Robert, Curran Marla, Kuranda Klaudia, Mingozzi Federico, Chang Tiffany, Reape Kathleen Z, Anguela Xavier M, High Katherine A
Abstract excerpt
BACKGROUND: The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. METHODS: In this phase 1-2 trial, we infused an investigational adeno-associated viral (AAV) vector (SPK-8011) for hepatocyte expression of factor VIII in 18 men with hemophilia A. Four dose cohorts...
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