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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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2021
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Original Article
Accesso online: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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