Fig. 3.
Fig. 3. Gene transfer by MPIN into PNH-phenotype hematopoietic cell lines restores PIG-A function. / (A) GPI-APs (CD55, CD59, CD48) and NGFR surface expression of PIG-A–deficient B-lymphoblastoid cell line JY5 transduced with MN or MPIN on day 39. (B) (C) GPI-AP (CD59) and NGFR surface expression of PIG-A–deficient K562 mutant cell line (panel B) and PIG-A–deficient B-lymphoblastoid cell line (TK-14−) established from a patient with PNH (panel C), transduced with MN or MPIN on day 21. (D) Detection of the region including PIG-A in MPIN-transduced cell lines (JY5, K562 mutant, and TK-14−) by PCR. DNA was isolated from transduced (MN or MPIN) or untransduced (mock) cell lines. 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.

Gene transfer by MPIN into PNH-phenotype hematopoietic cell lines restores PIG-A function.

(A) GPI-APs (CD55, CD59, CD48) and NGFR surface expression of PIG-A–deficient B-lymphoblastoid cell line JY5 transduced with MN or MPIN on day 39. (B) (C) GPI-AP (CD59) and NGFR surface expression of PIG-A–deficient K562 mutant cell line (panel B) and PIG-A–deficient B-lymphoblastoid cell line (TK-14−) established from a patient with PNH (panel C), transduced with MN or MPIN on day 21. (D) Detection of the region including PIG-A in MPIN-transduced cell lines (JY5, K562 mutant, and TK-14−) by PCR. DNA was isolated from transduced (MN or MPIN) or untransduced (mock) cell lines. 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.

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