Fig. 3: Contour plot showing the relationship between study confidence, P1, and P2 resulting from the 2-stage statistical model. | Gene Therapy

Fig. 3: Contour plot showing the relationship between study confidence, P1, and P2 resulting from the 2-stage statistical model.

From: Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Fig. 3

Results were generated with Eq. 1. P1 is the probability an F0 male acquires and transmits transgene DNA to an F1 offspring. P2 is the probability an F1 offspring inherits and retains the transgene DNA. P = P1 * P2 is the overall probability of a germline transmission; lines represent combinations of P1 * P2 that would result in P, probability of germline transmission, between 0.01 and 0.05. The colored regions represent the combinations of P1 and P2, covering various ranges of study confidence, using the 22 F0 males who received AAV5-hFVIII-SQ and their combined 139 F1 offspring.

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