Table 1 Study confidence that ≥1 hFVIII-SQ germline transmission event would have been detected given assumed probabilities for the risk of germline transmission.

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

Overall probability of germline transmission (P)

P1

P2

Confidence/power

0.050

0.316

0.158

0.992

0.040

0.283

0.141

0.982

0.030

0.245

0.122

0.954

0.020

0.200

0.100

0.884

0.015

0.173

0.087

0.810

  1. P = P1 * P2, where P1 is the assumed probability an F0 male acquires and transmits transgene DNA to an F1 offspring, and P2 is the assumed probability an F1 offspring inherits and retains the transgene DNA. Confidence values were calculated using Eq. 1 as stated in the Methods section and were based on the number of mice born to each of the 22 F0 males that received AAV5-hFVIII.
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