GSM4630048[Accession] - GEO DataSets

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Items: 4

Select item 2001531011.

Gene therapy of dominant CRX-Leber congenital amaurosis using patient retinal organoids

(Submitter supplied) This SuperSeries is composed of the SubSeries listed below.

Organism:: Homo sapiens

Type:: Expression profiling by high throughput sequencing

Platforms:: GPL16791 GPL24676
43 Samples

Download data: H5

Series

Accession:: GSE153101

ID:: 200153101

PubMed Full text in PMC Similar studies

Select item 2001529392.

Gene therapy of dominant CRX-Leber congenital amaurosis using patient retinal organoids I

(Submitter supplied) Mutations in the cone-rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Using induced pluripotent stem cells (iPSCs) from a patient with CRX-I138fs mutation, we established an in vitro model of CRX-LCA in retinal organoids that exhibit defective photoreceptor maturation by histology and gene profiling including diminished expression of visual opsins. more...