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Identifying Biomarkers of Heterogeneity and Transplantation Efficacy in Retinal Pigment Epithelial Cells [Bulk RNA-seq]

ABSTRACT: Identifying Biomarkers of Heterogeneity and Transplantation Efficacy in Retinal Pigment Epithelial Cells [Bulk RNA-seq]

PROVIDER: PRJNA869448 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR21046616_1.fastq.gz	Fastqsanger.gz
	SRR21046616_2.fastq.gz	Fastqsanger.gz
	SRR21046617_1.fastq.gz	Fastqsanger.gz
	SRR21046617_2.fastq.gz	Fastqsanger.gz
	SRR21046618_1.fastq.gz	Fastqsanger.gz

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Identifying biomarkers of heterogeneity and transplantation efficacy in retinal pigment epithelial cells

Project description:Transplantation of retinal pigment epithelial (RPE) cells holds great promise for patients with retinal degenerative diseases, such as age-related macular degeneration. In-depth characterization of RPE cell product identity and critical quality attributes are needed to enhance efficacy and safety of replacement therapy strategies. Here, we characterized an adult RPE stem cell-derived (RPESC-RPE) cell product using bulk and single-cell RNA sequencing (scRNA-seq), assessing functional cell integration in vitro into a mature RPE monolayer and in vivo efficacy by vision rescue in the Royal College of Surgeons rats. scRNA-seq revealed several distinct subpopulations in the RPESC-RPE product, some with progenitor markers. We identified RPE clusters expressing genes associated with in vivo efficacy and increased cell integration capability. Gene expression analysis revealed lncRNA (TREX) as a predictive marker of in vivo efficacy. TREX knockdown decreased cell integration while overexpression increased integration in vitro and improved vision rescue in the RCS rats.

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Identifying Biomarkers of Heterogeneity and Transplantation Efficacy in Retinal Pigment Epithelial Cells [Bulk RNA-seq]

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