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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Bibliografski detalji
Izdano u:Mol Ther Methods Clin Dev
Glavni autori: Depla, Josse A., Sogorb-Gonzalez, Marina, Mulder, Lance A., Heine, Vivi M., Konstantinova, Pavlina, van Deventer, Sander J., Wolthers, Katja C., Pajkrt, Dasja, Sridhar, Adithya, Evers, Melvin M.
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2020
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Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7327852/
https://ncbi.nlm.nih.gov/pubmed/32637448
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.05.028
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