Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

The development of gene therapies for central nervous system disorders is challenging because it is difficult to translate preclinical data from current in vitro and in vivo models to the clinic. Therefore, we developed induced pluripotent stem cell (iPSC)-derived cerebral organoids as a model for r...

Ausführliche Beschreibung

Bibliografische Detailangaben

Hauptverfasser:	Josse A. Depla, Marina Sogorb-Gonzalez, Lance A. Mulder, Vivi M. Heine, Pavlina Konstantinova, Sander J. van Deventer, Katja C. Wolthers, Dasja Pajkrt, Adithya Sridhar, Melvin M. Evers
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Elsevier 2020-09-01
Schriftenreihe:	Molecular Therapy: Methods & Clinical Development
Schlagworte:	Cerebral organoids AAV adeno-associated virus CNS central nervous system Transduction
Online-Zugang:	http://www.sciencedirect.com/science/article/pii/S2329050120301157
Tags:	Tag hinzufügen Keine Tags, Fügen Sie das erste Tag hinzu!