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Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates

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Giang N. Nguyen, Maria C. Seleme, Jonathan R. Lindgren, Allysen C. Henriksen, Amy Muehlmatt, Melanie McFadden, Geary R. Smith, Denise E. Sabatino
Journal: Blood
Blood (2022) 140 (Supplement 1): 1902–1903.
https://doi.org/10.1182/blood-2022-168340
Published: 2022