Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

Evaluation of immune responses to adeno-associated virus (AAV)-mediated gene therapies prior to and following dose administration plays a key role in determining therapeutic safety and efficacy. This report describes up to 3 years of immunogenicity data following administration of valoctocogene roxa...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Long, Brian R., Veron, Philippe, Kuranda, Klaudia, Hardet, Romain, Mitchell, Nina, Hayes, Gregory M., Wong, Wing Yen, Lau, Kelly, Li, Mingjin, Hock, M. Benjamin, Zoog, Stephen J., Vettermann, Christian, Mingozzi, Federico, Schweighardt, Becky
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7854299/
https://ncbi.nlm.nih.gov/pubmed/33309883
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.12.008
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