Fig. 7: Effects of single administration of AAV donor in vitro and in vivo.

a Pigo-KO Neuro2a cells were transfected with pAAVCas9 and pAAV donor (pAAV-mPigoHITIgRNA) or pAAV donor only and analyzed over time by FACS for measuring the restored population. % of CD24 positive cells were shown in the right graph. b Comparison of growth in HITI-treated (AAVCas9 and AAV donor) and AAV donor treated Pigo^b/− mice. Data are presented as mean values + SD. c Elevation of Gr-1, a GPI-AP, levels on blood granulocytes from Pigo^b/− mice at 4 months after HITI-treated or AAV donor treated Pigo^b/− mice. n number of animals, ns not significant. (*p < 0.05) (t-test, one-sided, P-value, KIKO:KIKO + HITI, 0.035; KIKO:KIKO + Pigo, 0.035) Data are presented as mean values + SEM. d Amelioration of hyperphosphatasia. (*p < 0.05; **p < 0.01) Data are presented as mean values + SEM. (t-test, two-sided, P-value, KIKO:KIKO + HITI, 5.5 × 10⁻³; KIKO:KIKO + Pigo, 0.029). e Improved performance in the hanging test with 5 mm (left) and 11 mm (right) meshes of AAV donor only treated Pigo^b/b mice in comparison with HITI-treated Pigo^b/b mice. Data are presented as mean values + SEM. (t-test, two-sided). f Severity scores of tremor appeared in AAV donor only treated Pigo^b/b mice and HITI treated Pigo^b/b mice at five months (t-test, one-sided, P-value, 0.034). Data are presented as mean values + SEM. Source data are provided as a Source Data file.