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Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype five gene therapy under investigation for the treatment of hemophilia A. Herein, we assessed the potential for germline transmission of AAV5-hFVIII-SQ in mice. Male B6.129S6-Rag2(tm1Fwa) N12 mice received a single intr...

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Detalles Bibliográficos
Autores principales:	Fonck, Carlos, Su, Cheng, Arens, Jeremy, Koziol, Elli, Srimani, Jaydeep, Henshaw, Joshua, Van Tuyl, Andrea, Chandra, Sundeep, Vettermann, Christian, O’Neill, Charles A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Brief Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10457182/ https://www.ncbi.nlm.nih.gov/pubmed/35132205 http://dx.doi.org/10.1038/s41434-022-00318-5

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