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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...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Veröffentlicht in:J Biomol Tech
Hauptverfasser: Alcudia, Javier, Wright, John Fraser
Format: Artigo
Sprache:Inglês
Veröffentlicht: Association of Biomolecular Resource Facilities 2020
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7424966/
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