Article

Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies

2018-10-16

Abstract excerpt

Human genetic disorders provide a powerful lens to understanding the human brain. Induced pluripotent stem cells (iPSC) represent an important, new resource for mechanistic studies and therapeutic development. Christianson syndrome (CS), an X-linked neurological disorder with attenuation of brain growth postnatally (postnatal microcephaly), is caused by mutations in SLC9A6, the gene encoding endosomal Na + /H +...

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Literature Corpus work: 4411e40b-afc8-520b-8533-f211b0cae380
DOI: 10.1101/444232

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