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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...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2022
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Materias: | |
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 |