title: CNS disease models with human pluripotent stem cells in the CRISPR age.
abstract: In vitro differentiation of human pluripotent stem cells provides a systematic platform to investigate the physiological development and function of the human nervous system, as well as the etiology and consequence when these processes go awry. Recent development in three-dimensional (3D) organotypic culture systems allows modeling of the complex structure formation of the human CNS, and the intricate interactions between various resident neuronal and glial cell types. Combined with an ever-expanding genome editing and regulation toolkit such as CRISPR/Cas9, it is now a possibility to study human neurological disease in the relevant molecular, cellular and anatomical context. In this article, we review recent progress in 3D neural culture and the implications for disease modeling.
authors: Whitehead Institute for Biomedical Research, 9Cambridge Center, Cambridge, MA 02142, United States.
authors: Julien
authors: J
authors: Muffat
authors: Whitehead Institute for Biomedical Research, 9Cambridge Center, Cambridge, MA 02142, United States.
authors: Yun
authors: Y
authors: Li
authors: Whitehead Institute for Biomedical Research, 9Cambridge Center, Cambridge, MA 02142, United States. Electronic address: jaenisch@wi.mit.edu.
authors: Rudolf
authors: R
authors: Jaenisch
mesh_terms: D000818:Animals; D002493:Central Nervous System Diseases / Q000378:metabolism / Q000473:pathology*; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D005818:Genetic Engineering; D006801:Humans; D008954:Models, Biological; D009940:Organoids / Q000473:pathology; D039904:Pluripotent Stem Cells / Q000166:cytology*