Retroviral vector MPIN restores PIG-A function in PNH-phenotype BMMCs.

(A) GPI-AP (CD59) and NGFR surface expression of the pooled colonies/bursts generated from PNH-phenotype BMMCs transduced with MN or MPIN. (B) Detection of the region including PIG-A in MPIN-transduced colonies/bursts generated from PNH-phenotype BMMCs by PCR. DNA was isolated from transduced (MN or MPIN) or untransduced (mock) colonies/bursts. A region including vector plus 5′PIG-A was amplified by PCR. For positive control, the MPIN vector plasmid was used as a template, and Marker 6 was loaded as a marker.