Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) vector-based gene therapy for spinal muscular atrophy (SMA). Patients with elevated titers of anti-AAV9 antibodies (AAV9-Ab) should not receive onasemnogene abeparvovec because of potential safety and efficacy implica...

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Detalles Bibliográficos
Autores principales: Day, John W., Mendell, Jerry R., Burghes, Arthur H.M., van Olden, Rudolf W., Adhikary, Rishi R., Dilly, Keith W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10562739/
https://www.ncbi.nlm.nih.gov/pubmed/37822718
http://dx.doi.org/10.1016/j.omtm.2023.101117

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10562739/
https://www.ncbi.nlm.nih.gov/pubmed/37822718
http://dx.doi.org/10.1016/j.omtm.2023.101117

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