Development challenges associated with rAAV-based gene therapies

The number of gene therapies in development continues to increase, as they represent a novel method to treat, and potentially cure, many diseases. Gene therapies can be conducted with an in vivo or ex vivo approach, to cause gene augmentation, gene suppression, or genomic editing. Adeno-associated v...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Journal of toxicological sciences 2021, Vol.46(2), pp.57-68
Hauptverfasser:	Bolt, Michael W., Brady, Joseph T., Whiteley, Lawrence O., Khan, K. Nasir
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV gene therapy Clinical trials Development challenges Gene therapy Genomics Hepatotoxicity Immunogenicity Insertional mutagenesis Neurotoxicity Nonclinical toxicity Viruses
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!