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Table 1 Expression of mouse endogenous CAG-containing genes after treatment with ZF-KOX1 and mZF-KRAB

From: Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

Treatment

Weeks post-injection

Linear regression

Htt (4,7) %

Linear regression

Atn1 (3,10) %

Linear regression

Atxn2 (6,10) %

Linear regression

Tbp (3,13) %

ZF-KOX1

2

R 2= 0.97 §

92.1 ± 0.4*

R 2 = 0.03

101.2 ± 13.8

R 2 = 0.09

95.5 ± 3.9

R 2= 0.98 §

94.5 ± 5.0

4

R 2= 0.99 §

89.6 ± 2.4*

R 2 = 0.64

91.7 ± 13.7

R 2 = 0.57

96.1 ± 6.8

R 2 = 0.98

91.5 ± 1.7

6

R 2= 0.99*

90.6 ± 9.6

R 2 = 0.09

106.3 ± 4.9

R 2 = 0.96

98.4 ± 2.9

R 2 = 0.65

98.1 ± 3.3

mZF-KRAB

2

R 2 = 0.53

98.3 ± 3.3

R 2 = 0.02

109.4 ± 10.6

R 2 = 0.00

103.3 ± 11.8

R 2 = 0.08

99.1 ± 7.9

4

R 2 = 0.14

88.4 ± 6.7

R 2 = 0.19

88.6 ± 7.3

R 2 = 0.36

89.6 ± 5.4

R 2 = 0.43

90.5 ± 10.4

6

R 2 = 0.43

92.5 ± 11.35

R 2 = 0.16

91.4 ± 6.5

R 2 = 0.00

96.7 ± 4.4

R 2 = 0.07

94.7 ± 4.8

  1. The first number (in brackets after the name of the gene) represents the number of CAG repeats, the second the number of glutamines in the coding stretch (CAG + CAA). Values are given as the percentage expression of the gene of interest, with respect to the average values in the control hemispheres. The result of ZF-KOX1, 6 weeks post-injection, should be taken with caution, since at this time point there was a significant neuronal loss and leakage of the vector to the contralateral hemisphere. In bold: § P < 0.1; *P < 0.05. Atn1: atrophin 1; Atxn2: ataxin 2; Htt: huntingtin (mouse); Tbp: TATA binding protein