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. 2022 Sep 2;27:47–60. doi: 10.1016/j.omtm.2022.08.009

Table 1.

Enrollment status

Subject ID Mutation At gene transfer
Age (years) Weight (kg) Dose
Subject 1 duplication exon 22–41 8.9 37.8 2.5 × 1013 vg/kg per leg (5.0 × 1013 vg/kg bilateral total)
Subject 2 nonsense mutation, exon 19 (c.2332C>T/p.Gln778Ter) 6.9 20.9 5.0 × 1013 vg/kg per leg (1.0 × 1014 vg/kg bilateral total)

Enrollment and dosage information for subjects 1 and 2. All AAV vector genome (vg) doses were relative to kilograms of total body weight.