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Article

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Genome research - 1 Sept 2014

Xie Fei, Ye Lin, Chang Judy C, Beyer Ashley I, Wang Jiaming, Muench Marcus O, Kan Yuet Wai

Abstract excerpt

β-thalassemia, one of the most common genetic diseases worldwide, is caused by mutations in the human hemoglobin beta (HBB) gene. Creation of human induced pluripotent stem cells (iPSCs) from β-thalassemia patients could offer an approach to cure this disease. Correction of the disease-causing mutations in iPSCs could restore normal function and provide a rich source of cells for transplantation. In this study,...

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Source

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Identifiers

PMID: 25096406
DOI: 10.1101/gr.173427.114
PMCID: PMC4158758

Genes

HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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