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.
|Number of pages||9|
|Journal||Molecular Therapy - Methods and Clinical Development|
|Publication status||Published - 11 Sep 2020|