Table 1.

Evaluation of Gene Transfer and Proviral Gene Expression Into Canine MPS I LTMC Adherent Layers and Individual CFU-GM Derived From Transduced LTMCs

Dogs Vectors Adherent Layer Cells Individual LTMC-Derived CFU-GM
Proviral PCR α-ID Activity RT-PCR Proviral PCR*α-ID Activity
M2  M48αID  Positive  >10 U† Positive  62%  17%  
 LCαIDSN    (13/21)  (5/30)  
M3  M48αID  Positive 101 U  Positive  27%   8% 
      (4/15)  (2/25)  
M4  M48αID Positive  84 U  Positive  20%  16% 
      (3/15)  (5/32)  
M5  M48αID Positive  198 U  Positive  24%  20% 
      (5/21)  (2/10)  
M6  LCαIDSN Positive  100 U  Positive  31%  ND  
 M48αID    (13/42)  
Untransduced control   Negative 0 U  Negative  0/20  0/12 
Dogs Vectors Adherent Layer Cells Individual LTMC-Derived CFU-GM
Proviral PCR α-ID Activity RT-PCR Proviral PCR*α-ID Activity
M2  M48αID  Positive  >10 U† Positive  62%  17%  
 LCαIDSN    (13/21)  (5/30)  
M3  M48αID  Positive 101 U  Positive  27%   8% 
      (4/15)  (2/25)  
M4  M48αID Positive  84 U  Positive  20%  16% 
      (3/15)  (5/32)  
M5  M48αID Positive  198 U  Positive  24%  20% 
      (5/21)  (2/10)  
M6  LCαIDSN Positive  100 U  Positive  31%  ND  
 M48αID    (13/42)  
Untransduced control   Negative 0 U  Negative  0/20  0/12 

Abbreviation: ND, sample not done.

*

CFU positive by PCR amplification of proviral neor or α-ID sequences.

†

Units are nanomoles per milligram per hour (normal activity ∼1 U).

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