Table 1 Hematological parameters in Townes-SCD mice.

From: Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

 

% hHgbA

RBC (×106/µl)

HGB (g/dl)

% Retics

PLT (×103/µl)

WBC (×103/µl)

% myeloid HBB HR correction

% myeloid HBB INDELS

Pre-HSCT (n = 7)

0

7.0 ± 0.8

11.5 ± 1.1

60.7 ± 4.6

756 ± 159

61.1 ± 16.4

N/A

N/A

HbA into HbS (n = 4)

100

9.4 ± 1.1

11.6 ± 0.6

8.6 ± 0.4

949 ± 230

13.6 ± 8.9

N/A (100% HbA)

N/A (100% HbA)

HbS into HbS (n = 2)

0

6.4

10

51.5

1264

58.7

N/A (100% HbS)

N/A (100% HbS)

Mouse A

84.1

7.18

8.9

22.3

1380

18.3

35.4

9.6

Mouse B

95.1

6.55

8.3

17.5

1391

13.9

22.4

21.0

Mouse C

33.0

4.05

6.9

53.5

931

53

1.0

82.6

Mouse D

58.2

4.39

6.9

48.5

716

50.8

11.1

79.5

Mouse E

59.6

3.23

5.6

51.4

737

51.7

8.2

81.2

Mouse F

70.4

4.96

7.2

37.3

851

33.8

19.3

68.3

Mouse G

70.7

4.74

7

33.7

881

21.3

16.5

71.2

Mouse H

15.5

3.37

5.7

57.9

473

61.2

1.5

79.5

Mouse I

65.0

3.83

5.6

33.7

1307

44.2

17.5

63.0

  1. Hematological parameters for pre-transplanted Townes-SCD HbS mice, 16-week post-transplantation for HbS recipients, and 16-week post-transplantation for individual recipients of HBB gene-corrected Townes-SCD HSCs. In all cases, recipient mice were HbS Townes-SCD mice. Where displayed, averages are mean ± standard deviation. Retics %, percentage of PB reticulocytes.
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