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& Happle, R

However, percent changes from baseline appeared comparable to those observed in healthy subjects (Fig

J Lipid Res (2004) 45(8):137597

Beti-cel is generated by ex vivo transduction of autologous CD34 + hematopoietic stem and progenitor cells HSPC) with the lentiviral vector BB305, which is a replication defective, self-inactivating (SIN), third-generation HIV-1 based lentiviral vector engineered to deliver a variant of the human -globin gene modified at codon 87 (T87Q) (Fig