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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