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Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides

Vila-Julià, Ferran, Cabrera-Pérez, Raquel, Cámara, Yolanda, Molina-Berenguer, Miguel, Lope-Piedrafita, Silvia, Hirano, Michio, Mingozzi, Federico, Torres-Torronteras, Javier, Martí, Ramon·2020·eBioMedicine
DOI 10.1016/j.ebiom.2020.103133PMC PMC7689515
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Fig. 1 Effect of AAV treatment on dThd pharmacokinetics. a) Plasma dThd concentration in WT and dKO mice exposed to dThd and dUrd supplementation in drinking water starting from weaning (exposed group), or without exposure (naïve group). The monitored range encompasses 5 to 34 weeks of age. Each symbol and line represents a single mouse. Gray dotted and shadowed areas indicate the concentration range in naïve dKO and WT groups, respectively. b) Plasma dThd concentrations before (0 h) and after a single administration of dThd and dUrd by oral gavage (166 mg/kg each) in naïve and exposed WT mice, naïve and exposed dKO mice, and exposed dKO mice previously treated with different doses of AAV-TBG, AAV-AAT and AAV-HLP vectors. Mice were 35 to 45 weeks old at the time of the experiment. Each point represents mean±range. White and gray areas between dotted lines indicate the concentration range in naïve dKO and WT groups at time zero, respectively. c, d and e) PK results (mean+SD) obtained from experiments depicted at b. Sample sizes in panels b to e are: naïve WT, n = 8; naïve dKO, n = 6; exposed WT, n = 5; exposed dKO, n = 7; TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 5, and 1013 vg/kg n = 5; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 5; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; panels c and d, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed; panel e, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with each other). AUC, area under the curve.
Fig. 1 Effect of AAV treatment on dThd pharmacokinetics. a) Plasma dThd concentration in WT and dKO mice exposed to dThd and dUrd supplementation in drinking water starting from weaning (exposed group), or without exposure (naïve group). The monitored range encompasses 5 to 34 weeks of age. Each symbol and line represents a single mouse. Gray dotted and shadowed areas indicate the concentration range in naïve dKO and WT groups, respectively. b) Plasma dThd concentrations before (0 h) and after a single administration of dThd and dUrd by oral gavage (166 mg/kg each) in naïve and exposed WT mice, naïve and exposed dKO mice, and exposed dKO mice previously treated with different doses of AAV-TBG, AAV-AAT and AAV-HLP vectors. Mice were 35 to 45 weeks old at the time of the experiment. Each point represents mean±range. White and gray areas between dotted lines indicate the concentration range in naïve dKO and WT groups at time zero, respectively. c, d and e) PK results (mean+SD) obtained from experiments depicted at b. Sample sizes in panels b to e are: naïve WT, n = 8; naïve dKO, n = 6; exposed WT, n = 5; exposed dKO, n = 7; TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 5, and 1013 vg/kg n = 5; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 5; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; panels c and d, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed; panel e, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with each other). AUC, area under the curve.CC BY-NC-ND · PubMed Central
Fig. 2 Effect of AAV treatment on dThd levels in 22-month-old mice. Plasma, liver, brain, and small intestine dThd concentration in WT (n = 7–9) and dKO (n = 8) naïve mice, WT (n = 4) and dKO (n = 10) exposed mice, and dKO exposed mice treated with different AAV doses (TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Horizontal lines indicate the median concentration for each group. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).
Fig. 2 Effect of AAV treatment on dThd levels in 22-month-old mice. Plasma, liver, brain, and small intestine dThd concentration in WT (n = 7–9) and dKO (n = 8) naïve mice, WT (n = 4) and dKO (n = 10) exposed mice, and dKO exposed mice treated with different AAV doses (TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Horizontal lines indicate the median concentration for each group. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).CC BY-NC-ND · PubMed Central
Fig. 3 TP activity in liver after AAV treatment. Liver TP activity in (a) WT (n = 8) and dKO (n = 8) naïve mice and in untreated WT (n = 4) and dKO (n = 10) exposed mice. (b) Liver TP activity in untreated WT and dKO exposed mice and in dKO exposed mice treated with different AAVs doses (TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). (c) Liver vector copy number (VCN) in AAV-treated dKO exposed mice. TBG-treated mice with 5 × 1011 vg/kg n = 1, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 3. Horizontal lines indicate the median for each group. (d) Non-linear regression of liver TP activity (data from panel b) vs. liver VCN (data from panel c) in AAV-treated mice (Y = intercept + slope x log(X)). Note that the low VCN region is magnified to facilitate observation of the differences between the three vectors. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; for panel a, Mann-Whitney U test; for panel b, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with each other, two different analyses for each dose).
Fig. 3 TP activity in liver after AAV treatment. Liver TP activity in (a) WT (n = 8) and dKO (n = 8) naïve mice and in untreated WT (n = 4) and dKO (n = 10) exposed mice. (b) Liver TP activity in untreated WT and dKO exposed mice and in dKO exposed mice treated with different AAVs doses (TBG-treated mice with 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). (c) Liver vector copy number (VCN) in AAV-treated dKO exposed mice. TBG-treated mice with 5 × 1011 vg/kg n = 1, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 4; AAT-treated mice with 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 3. Horizontal lines indicate the median for each group. (d) Non-linear regression of liver TP activity (data from panel b) vs. liver VCN (data from panel c) in AAV-treated mice (Y = intercept + slope x log(X)). Note that the low VCN region is magnified to facilitate observation of the differences between the three vectors. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; for panel a, Mann-Whitney U test; for panel b, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with each other, two different analyses for each dose).CC BY-NC-ND · PubMed Central
Fig. 4 Motor function. Motor function assessed by rotarod test in 25-week-old WT and dKO naïve and exposed mice, and dKO exposed mice treated with different AAVs doses. Horizontal lines show median time to fall in each group. Gray areas indicate the range of values in the naïve WT group. No statistical differences found, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).
Fig. 4 Motor function. Motor function assessed by rotarod test in 25-week-old WT and dKO naïve and exposed mice, and dKO exposed mice treated with different AAVs doses. Horizontal lines show median time to fall in each group. Gray areas indicate the range of values in the naïve WT group. No statistical differences found, Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).CC BY-NC-ND · PubMed Central
Fig. 5 Brain MRI analysis. (a) Coronal T2-weighted and T2 map images of 1-mm sections (region corresponding to slice 6 in Supp Figure S7) showing ventricular enlargement in dKO exposed mice (red arrows). (b) Ventricular volume in 80 to 84-weeks old WT (n = 9) and dKO (n = 5) naïve mice, non-treated WT (n = 5) and dKO (n = 12) exposed mice, and dKO exposed mice treated with different AAVs doses (TBG-treated mice with: 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 3, 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 5; AAT-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Ventricular volume was quantified in T2 maps as the sum of the ventricular volumes calculated in all sections analyzed in Supp Figure S6 (see materials and methods for calculations). Gray areas indicate the range of values in the naïve WT group. Asterisks indicate statistically significant differences (*p<0.05; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed). Ventricular volume values above the mean plus 3 SD of all data were considered outliers and excluded from the analysis. Two outliers were identified in dKO exposed AVV-treated with TBG 1012 vg/kg, and TBG 2 × 1012 vg/kg, one in each group.
Fig. 5 Brain MRI analysis. (a) Coronal T2-weighted and T2 map images of 1-mm sections (region corresponding to slice 6 in Supp Figure S7) showing ventricular enlargement in dKO exposed mice (red arrows). (b) Ventricular volume in 80 to 84-weeks old WT (n = 9) and dKO (n = 5) naïve mice, non-treated WT (n = 5) and dKO (n = 12) exposed mice, and dKO exposed mice treated with different AAVs doses (TBG-treated mice with: 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 3, 2 × 1012 vg/kg n = 5 and 1013 vg/kg n = 5; AAT-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Ventricular volume was quantified in T2 maps as the sum of the ventricular volumes calculated in all sections analyzed in Supp Figure S6 (see materials and methods for calculations). Gray areas indicate the range of values in the naïve WT group. Asterisks indicate statistically significant differences (*p<0.05; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed). Ventricular volume values above the mean plus 3 SD of all data were considered outliers and excluded from the analysis. Two outliers were identified in dKO exposed AVV-treated with TBG 1012 vg/kg, and TBG 2 × 1012 vg/kg, one in each group.CC BY-NC-ND · PubMed Central
Fig. 6 Mitochondrial dNTPs. Mitochondrial thymidine triphosphate (dTTP) and deoxycytidine triphosphate (dCTP) content in liver (a and c) and brain (b and d) of WT (n = 9) and dKO (n = 8) naïve mice, non-treated WT (n = 4) and dKO (n = 10) exposed mice and dKO exposed mice treated with different AAVs doses (TBG-treated mice with: 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Horizontal lines indicate the median for each group. Gray areas indicate the range of values in the naïve WT group. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).
Fig. 6 Mitochondrial dNTPs. Mitochondrial thymidine triphosphate (dTTP) and deoxycytidine triphosphate (dCTP) content in liver (a and c) and brain (b and d) of WT (n = 9) and dKO (n = 8) naïve mice, non-treated WT (n = 4) and dKO (n = 10) exposed mice and dKO exposed mice treated with different AAVs doses (TBG-treated mice with: 5 × 1011 vg/kg n = 2, 1012 vg/kg n = 4, 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; AAT-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 4; HLP-treated mice with: 2 × 1012 vg/kg n = 6 and 1013 vg/kg n = 3). Horizontal lines indicate the median for each group. Gray areas indicate the range of values in the naïve WT group. Asterisks indicate statistically significant differences (*p<0.05, **p<0.01; Kruskal-Wallis test followed by the Dunn's multiple comparisons test, all groups compared with dKO exposed).CC BY-NC-ND · PubMed Central

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Authors
Vila-Julià, F., Cabrera-Pérez, R., Cámara, Y., Molina-Berenguer, M., Lope-Piedrafita, S., Hirano, M., Mingozzi, F., Torres-Torronteras, J., & Martí, R.
Venue
eBioMedicine
Publisher
Elsevier BV
Published
2020-12
Volume
62
Pages
103133
ISSN
2352-3964
DOI
10.1016/j.ebiom.2020.103133
PMC
PMC7689515
Cited by
22
References
5
Language
en
Licence
Funders
  • Generalitat de Catalunya (2018FI_B_01115, SLT002/16/00370)
  • Instituto de Salud Carlos III (PI15/00465, PI18/01574, PMP15/00025)

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