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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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Detalles Bibliográficos
Autores principales:	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.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7327852/ https://www.ncbi.nlm.nih.gov/pubmed/32637448 http://dx.doi.org/10.1016/j.omtm.2020.05.028

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