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Article

Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells.

The Journal of biological chemistry - 5 Aug 2016

Niu Xiaohua, He Wenyin, Song Bing, Ou Zhanhui, Fan Di, Chen Yuchang, Fan Yong, Sun Xiaofang

Abstract excerpt

β-Thalassemia (β-Thal) is one of the most common genetic diseases in the world. The generation of patient-specific β-Thal-induced pluripotent stem cells (iPSCs), correction of the disease-causing mutations in those cells, and then differentiation into hematopoietic stem cells offers a new therapeutic strategy for this disease. Here, we designed a CRISPR/Cas9 to specifically target the Homo sapiens hemoglobin β...

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Identifiers

PMID: 27288406
DOI: 10.1074/jbc.M116.719237
PMCID: PMC4974373

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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