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Article

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs.

Scientific reports - 9 Jul 2015

Xu Peng, Tong Ying, Liu Xiu-zhen, Wang Ting-ting, Cheng Li, Wang Bo-yu, Lv Xiang, Huang Yue, Liu De-pei

Abstract excerpt

β-Thalassemia is one of the most common genetic blood diseases and is caused by either point mutations or deletions in the β-globin (HBB) gene. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations may be a potential therapeutic strategy for this disease. Due to the low efficiency of typical homologous recombination, endonucleases,...

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Source

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Identifiers

PMID: 26156589
DOI: 10.1038/srep12065
PMCID: PMC4496796

Genes

HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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