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...

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מידע ביבליוגרפי

Principais autores:	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
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	Elsevier 2020-09-01
סדרה:	Molecular Therapy: Methods & Clinical Development
נושאים:	Cerebral organoids AAV adeno-associated virus CNS central nervous system Transduction
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2329050120301157
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