Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.
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A. Nienhuis | J. Gray | M. Valentine | A. Davidoff | J. Allay | R. Donahue | K. High | S. Waddington | F. Mingozzi | M. B. McCarville | A. Nathwani | J. Mcintosh | C. Ng | Roderick Bronson | Junfang Zhou | Yunyu Spence | D. Raj | G. Rastegarlari | Scott Jackson | S. Sleep | C. Rosales | D. Nathwani | S. Nawathe | J. Coleman | J. McIntosh | C. Ng | Cecilia Rosales