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Klamroth, Robert; Hayes, Gregory; Andreeva, Tatiana; Gregg, Keith; Suzuki, Takashi; Mitha, Ismail Haroon; Hardesty, Brandon; Shima, Midori; Pollock, Toni; Slev, Patricia; Oldenburg, Johannes; Ozelo, Margareth C; Stieltjes, Natalie; Castet, Sabine-Marie; Mahlangu, Johnny; Peyvandi, Flora; Kazmi, Rashid; Schved, Jean-François; Leavitt, Andrew D; Callaghan, Michael; Pan-Petesch, Brigitte; Quon, Doris V; Andrews, Jayson; Trinh, Alex; Li, Mingjin; Wong, Wing Yen
Human gene therapy 33, Številka: 7-8Journal Article
Adeno-associated virus (AAV)-mediated gene therapy may provide durable protection from bleeding events and reduce treatment burden for people with hemophilia A (HA). However, pre-existing immunity against AAV may limit transduction efficiency and hence treatment success. Global data on the prevalence of AAV serotypes are limited. In this global, prospective, noninterventional study, we determined the prevalence of pre-existing immunity against AAV2, AAV5, AAV6, AAV8, and AAVrh10 among people ≥12 years of age with HA and residual FVIII levels ≤2 IU/dL. Antibodies against each serotype were detected using validated, electrochemiluminescent-based enzyme-linked immunosorbent assays. To evaluate changes in antibody titers over time, 20% of participants were retested at 3 and 6 months. In total, 546 participants with HA were enrolled at 19 sites in 9 countries. Mean (standard deviation) age at enrollment was 36.0 (14.87) years, including 12.5% younger than 18 years, and 20.0% 50 years of age and older. On day 1, global seroprevalence was 58.5% for AAV2, 34.8% for AAV5, 48.7% for AAV6, 45.6% for AAV8, and 46.0% for AAVrh10. Considerable geographic variability was observed in the prevalence of pre-existing antibodies against each serotype, but AAV5 consistently had the lowest seroprevalence across the countries studied. AAV5 seropositivity rates were 51.8% in South Africa ( = 56), 46.2% in Russia ( = 91), 40% in Italy ( = 20), 37.2% in France ( = 86), 26.8% in the United States ( = 71), 26.9% in Brazil ( = 26), 28.1% in Germany ( = 89), 29.8% in Japan ( = 84), and 5.9% in the United Kingdom ( = 17). For all serotypes, seropositivity tended to increase with age. Serostatus and antibody titer were generally stable over the 6-month sampling period. As clinical trials of AAV-mediated gene therapies progress, data on the natural prevalence of antibodies against various AAV serotypes may become increasingly important.
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Dostop do baze podatkov JCR je dovoljen samo uporabnikom iz Slovenije. Vaš trenutni IP-naslov ni na seznamu dovoljenih za dostop, zato je potrebna avtentikacija z ustreznim računom AAI.
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JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
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Povezave do osebnih bibliografij avtorjev | Povezave do podatkov o raziskovalcih v sistemu SICRIS |
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Vir: Osebne bibliografije
in: SICRIS
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