Table 1 Percentage of body weight and body length when compared to wildtype mice at corresponding sex and age.

From: First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model

 

Percentage of normal wildtype as 100% (at 16-week post AAV injection or equivalent to 20 to 21-week-old of mice age)

Uninjected control

AAV8 dose: 4 × 1010 vg/mouse

GHR+/+

GHR+/−

GHR−/−

AAV8-HLP-Luc

AAV8-HLP-mGHR

Mean

±SEM

Mean

±SEM

Mean

±SEM

Mean

±SEM

Mean

±SEM

Male

  Body weight

100.0

2.3

94.6

2.9

42.9

1.2

47.7

2.6

59.5*

3.3

  Body length

100.0

1.0

98.9

1.0

73.3

0.5

73.0

0.9

78.7***

0.8

Female

  Body weight

100.0

1.2

89.9

2.4

50.1

2.7

54.8

4.1

65.6n.s.

4.6

  Body length

100.0

1.1

98.0

0.9

73.7

0.6

76.0

0.9

81.1*

1.5

  1. n.s. P > 0.05; *P < 0.05; ***P < 0.001 Student’s unpaired t-test comparing values between AAV8-HLP-Luc and AAV8-HLP-mGHR.