Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated virus serotype 9 vector-based gene therapy th...

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Detalles Bibliográficos
Autores principales: Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7973120/
https://www.ncbi.nlm.nih.gov/pubmed/33768131
http://dx.doi.org/10.1016/j.omtm.2021.02.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7973120/
https://www.ncbi.nlm.nih.gov/pubmed/33768131
http://dx.doi.org/10.1016/j.omtm.2021.02.014

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