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

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Cerebral organoids
AAV
adeno-associated virus
CNS
central nervous system
Transduction
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301157

Internet

http://www.sciencedirect.com/science/article/pii/S2329050120301157

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