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One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system.

Stem cell research & therapy - 26 Feb 2018

Wattanapanitch Methichit, Damkham Nattaya, Potirat Ponthip, Trakarnsanga Kongtana, Janan Montira, U-Pratya Yaowalak, Kheolamai Pakpoom, Klincumhom Nuttha, Issaragrisil Surapol

Abstract excerpt

BACKGROUND: Thalassemia is the most common genetic disease worldwide; those with severe disease require lifelong blood transfusion and iron chelation therapy. The definitive cure for thalassemia is allogeneic hematopoietic stem cell transplantation, which is limited due to lack of HLA-matched donors and the risk of post-transplant complications. Induced pluripotent stem cell (iPSC) technology offers prospects for...

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Identifiers

PMID: 29482624
DOI: 10.1186/s13287-018-0779-3
PMCID: PMC5828150

Genes

HBB

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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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