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Research has shown approximately 40% oral bioavailability in animal studies
To ensure efficacy, specific administration guidelines have been established
N, 15.79
Hye Ok Kim, orcid.org/0000-0001-6957-6823
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
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