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Article

The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice.

Scientific reports - 1 Sept 2016

Ou Zhanhui, Niu Xiaohua, He Wenyin, Chen Yuchang, Song Bing, Xian Yexing, Fan Di, Tang Daolin, Sun Xiaofang

Abstract excerpt

β-thalassemia results from point mutations or small deletions in the β-globin (HBB) gene that ultimately cause anemia. The generation of induced pluripotent stem cells (iPSCs) from the somatic cells of patients in combination with subsequent homologous recombination-based gene correction provides new approaches to cure this disease. CRISPR/Cas9 is a genome editing tool that is creating a buzz in the scientific...

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Source

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Identifiers

PMID: 27581487
DOI: 10.1038/srep32463
PMCID: PMC5007518

Genes

HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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