Table 1.

Summary of CD34+ enrichment, CFU transduction efficiency, cell expansion, and engraftment kinetics of animals

Cytokine regimenVectorCD34+
enrichment*
Transduction
efficiency
Initial no.of cellsNo. of cells
at the end
of culture
ExpNo. of
infused
cells/kg
Eng1-153 (d)F/U1-155 (mo)
Animal RQ2314        18 
 G-CSF G1Na 100 89%  (25 of 28) 1.4 × 107 2.6 × 107 1.8 6.2 × 106   
 G-CSF+SCF LNL6 151 96%  (27 of 28) 2.1 × 107 2.6 × 108 12.4 6.2 × 107   
Animal RQ2284        15 
 G-CSF+SCF G1Na 73 100%  (16 of 16) 3.0 × 107 3.5 × 107 1.2 7.9 × 106   
 G-CSF LNL6 93 90%  (27 of 30) 2.2 × 107 2.2 × 107 1.0 4.8 × 106   
Animal RQ2237        12 
 G-CSF LNL6 113 100%  (32 of 32) 1.9 × 107 2.4 × 107 1.3 4.9 × 106   
 G-CSF+SCF G1Na 95 100%  (32 of 32) 4.5 × 107 1.1 × 108 2.5 2.2 × 107   
Animal RQ2277        12 
 G-CSF+SCF G1Na 155 100%  (32 of 32) 4.0 × 107 2.1 × 108 5.2 5.0 × 107   
 G-CSF+Flt3-L LNL6 95 100%  (32 of 32) 1.6 × 107 3.1 × 107 1.9 7.3 × 106   
Animal RQ2265        14 12 
 G-CSF+Flt3-L G1Na 127 100%  (32 of 32) 2.9 × 107 4.6 × 107 1.6 1.0 × 107   
 G-CSF+SCF LNL6 72 100%  (32 of 32) 3.9 × 107 9.2 × 107 2.3 2.1 × 107   
Cytokine regimenVectorCD34+
enrichment*
Transduction
efficiency
Initial no.of cellsNo. of cells
at the end
of culture
ExpNo. of
infused
cells/kg
Eng1-153 (d)F/U1-155 (mo)
Animal RQ2314        18 
 G-CSF G1Na 100 89%  (25 of 28) 1.4 × 107 2.6 × 107 1.8 6.2 × 106   
 G-CSF+SCF LNL6 151 96%  (27 of 28) 2.1 × 107 2.6 × 108 12.4 6.2 × 107   
Animal RQ2284        15 
 G-CSF+SCF G1Na 73 100%  (16 of 16) 3.0 × 107 3.5 × 107 1.2 7.9 × 106   
 G-CSF LNL6 93 90%  (27 of 30) 2.2 × 107 2.2 × 107 1.0 4.8 × 106   
Animal RQ2237        12 
 G-CSF LNL6 113 100%  (32 of 32) 1.9 × 107 2.4 × 107 1.3 4.9 × 106   
 G-CSF+SCF G1Na 95 100%  (32 of 32) 4.5 × 107 1.1 × 108 2.5 2.2 × 107   
Animal RQ2277        12 
 G-CSF+SCF G1Na 155 100%  (32 of 32) 4.0 × 107 2.1 × 108 5.2 5.0 × 107   
 G-CSF+Flt3-L LNL6 95 100%  (32 of 32) 1.6 × 107 3.1 × 107 1.9 7.3 × 106   
Animal RQ2265        14 12 
 G-CSF+Flt3-L G1Na 127 100%  (32 of 32) 2.9 × 107 4.6 × 107 1.6 1.0 × 107   
 G-CSF+SCF LNL6 72 100%  (32 of 32) 3.9 × 107 9.2 × 107 2.3 2.1 × 107   
*

Degree of progenitor cell enrichment by CD34 selection was calculated by enumerating total CFUs before and after column purification.

Transduction efficiency calculated by the percentage ofβ-actin-positive colonies that were also neopositive at the end of transduction, as determined by PCR of individual colonies.

Expansion: ratio of the number of cells at the end of 4 days of transduction (number of infused cells) to the number of cells plated on the first day.

F1-153

Engraftment: days to reach absolute neutrophil count greater than 500/μL.

F1-155

Follow-up: duration (months) of follow-up after transplantation.

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