AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.

colony dogs with factor levels in the 1%–5% range following gene therapy showed a trend toward higher rates in companion dogs (0.9 vs. 0.1, respectively) despite relatively similar cumulative follow-up duration (59 vs. 85 or 60 dog-years, respectively).

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Except for loss of balance and falling, which improved but did not reach statistical significance, all other negative attributes improved compared with pre-gene therapy baseline ( Figure 3 ).

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