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ABSTRACT: Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

PROVIDER: PRJNA891670 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR21985700_1.fastq.gz	Fastqsanger.gz
	SRR21985700_2.fastq.gz	Fastqsanger.gz
	SRR21985800_1.fastq.gz	Fastqsanger.gz
	SRR21985800_2.fastq.gz	Fastqsanger.gz
	SRR21985900_1.fastq.gz	Fastqsanger.gz

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Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

Project description:We have developed a therapeutic strategy for beta-hemoglobinopathies aimed at reactivating fetal hemoglobin expression in red blood cells derived from human hematopoietic stem/progenitor cells edited with CRISPR/Cas9 nucleases, cytidine or adenine base editors targeting the fetal gamma-globin promoters. Here, we report the transcriptomic changes occurring in human hematopoietic stem/progenitor cells (obtained from healthy donors) 48 h after transfection with CRISPR/Cas9 nucleases, cytidine or adenine base editors.

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