Table 1.

Human and porcine fVIII expression from genetically modified MSCs


Transgene

DNA copy no., fVIII transgenes per diploid genome equivalent

FVIII transcripts per cell*

FVIII activity, milliunits/24 h
Human fVIII   0.15  140   14  
Porcine fVIII
 
0.18§
 
116
 
84
 

Transgene

DNA copy no., fVIII transgenes per diploid genome equivalent

FVIII transcripts per cell*

FVIII activity, milliunits/24 h
Human fVIII   0.15  140   14  
Porcine fVIII
 
0.18§
 
116
 
84
 

MSCs were transduced with MSCV-human fVIII or MSCV-porcine fVIII at equal MOI and analyzed for gene marking, viral RNA levels, and fVIII production by qPCR, qRT-PCR, and chromogenic fVIII activity assay.

*

n = 3.

n = 6.

n = 2.

§

n = 3.

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