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

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*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Induced pluripotent stem cell-derived cerebral organoids are an innovative human model of the brain that could improve translation of gene therapy to the clinic. Depla et al. demonstrate the application of cerebral organoids as a preclinical model for AAV capsid selection and testing efficacy of therapeutic transgenes.

Original languageEnglish
Pages (from-to)167-175
Number of pages9
JournalMolecular Therapy - Methods and Clinical Development
Volume18
DOIs
Publication statusPublished - 11 Sep 2020

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