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Viral vector manufacturing: quality attributes of rAAV used in clinical development

Recombinant Adeno-Associated Virus (rAAV) are widely used for human gene therapy, now the basis for two licensed products (for RPE65-/-, and SMA) and numerous other investigational treatments for a range of diseases including hemophilia, cystic fibrosis, muscular dystrophy & many neurological di...

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Detalhes bibliográficos
Publicado no:J Biomol Tech
Main Authors: Alcudia, Javier, Wright, John Fraser
Formato: Artigo
Idioma:Inglês
Publicado em: Association of Biomolecular Resource Facilities 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7424686/
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