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Gene therapy: Practical aspects of implementation

The first wave of gene therapies for haemophilia submitted for regulatory review utilize a liver‐directed approach in which a functional gene copy of factor VIII (FVIII) or factor IX (FIX) is packaged inside a recombinant adeno‐associated viral vector (rAAV). Following a single treatment event, thes...

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Detalles Bibliográficos
Autores principales:	Pipe, Steven W., Reddy, K. Rajender, Chowdary, Pratima
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Supplement Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9324089/ https://www.ncbi.nlm.nih.gov/pubmed/35521727 http://dx.doi.org/10.1111/hae.14545

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