Figure 5 | Gene Therapy

Figure 5

From: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Figure 5

AAV2/5-OPTIRPE65 mediates physiological expression levels of RPE65 mRNA in the mouse retina. Whole retina lysates from WT mice 6 weeks following injection with AAV2/5-OPTIRPE65 or AAV2/2-hRPE65 were used for quantitative real-time PCR (qPCR). Absolute quantification of total mRNA-derived cDNA was performed for human RPE65 transcripts, OptiRPE65 transcripts and endogenous murine Rpe65 transcripts (mean±s.d.). Dose: 4 × 109 vg per eye, n=4.

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