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

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

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

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