Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy
| Autor: | Natalie L. Goedeker, Sachi D. Dharia, Danielle A. Griffin, Jesantha Coy, Todd Truesdale, Rajan Parikh, Kasen Whitehouse, Sourav Santra, Damon R. Asher, Craig M. Zaidman |
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| Jazyk: | angličtina |
| Rok vydání: | 2023 |
| Předmět: | |
| Zdroj: | Therapeutic Advances in Neurological Disorders, Vol 16 (2023) |
| Druh dokumentu: | article |
| ISSN: | 1756-2864 17562864 |
| DOI: | 10.1177/17562864221149781 |
| Popis: | Background: Adeno-associated virus (AAV) vectors are a promising platform for in vivo transfer of transgenes designed to treat diseases. Pre-existing humoral immunity to these vectors can potentially impact the safety and efficacy of gene therapies. Consequently, individuals with pre-existing antibodies to the specific AAV serotypes used may be excluded from clinical trials and treatments. Recombinant AAV serotype rh74 (rAAVrh74), a vector originally isolated from rhesus monkeys and potentially less immunogenic than other serotypes isolated from humans (e.g. AAV2, AAV5, and AAV9), efficiently transduces muscle and is being investigated for use in gene therapy for Duchenne muscular dystrophy (DMD). Objective: To evaluate prevalence of total binding antibodies (neutralizing and non-neutralizing) against rAAVrh74 in patients with DMD. Methods: Eligible individuals ( N = 107) were ⩾ 4 to |
| Databáze: | Directory of Open Access Journals |
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