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

Nathwani, Amit C, Rosales, Cecilia, McIntosh, Jenny, Rastegarlari, Ghasem, Nathwani, Devhrut, Raj, Deepak, Nawathe, Sushmita, Waddington, Simon N +16 more·2011·Molecular Therapy
DOI 10.1038/mt.2010.274

Cited by (345)

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