Fig. 5: Long-term partial rescue of severe MSUD phenotype in Bckdha−/− mice injected with AAV8-hAAT-hBCKDHA at 1014 vg/kg. | Nature Communications

Fig. 5: Long-term partial rescue of severe MSUD phenotype in Bckdha−/− mice injected with AAV8-hAAT-hBCKDHA at 1014 vg/kg.

From: Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

Fig. 5

a Weight curves for males (Bckdha−/− n = 4, Bckdha+/− n = 6, Bckdha+/+ n = 2) and b females (Bckdha−/− n = 2, Bckdha+/− n = 5 Bckdha+/+ n = 4). c Leucine and alloisoleucine concentrations in plasma until age 6 months (Bckdha−/− n = 6, Bckdha+/− n = 11, Bckdha+/+ n = 6, all injected). One Bckdha−/− male mouse died on day 28 and was not included for the points >day 28. All data are shown as mean ± SD. Source data are provided as a Source data file.

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