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Article

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.

Nature medicine - 1 Sept 2016

Traxler Elizabeth A, Yao Yu, Wang Yong-Dong, Woodard Kaitly J, Kurita Ryo, Nakamura Yukio, Hughes Jim R, Hardison Ross C, Blobel Gerd A, Li Chunliang, Weiss Mitchell J

Abstract excerpt

Disorders resulting from mutations in the hemoglobin subunit beta gene (HBB; which encodes β-globin), mainly sickle cell disease (SCD) and β-thalassemia, become symptomatic postnatally as fetal γ-globin expression from two paralogous genes, hemoglobin subunit gamma 1 (HBG1) and HBG2, decreases and adult β-globin expression increases, thereby shifting red blood cell (RBC) hemoglobin from the fetal (referred to as...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 27525524
DOI: 10.1038/nm.4170
PMCID: PMC5706766

Genes

SCD
HBG1
HBG2
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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A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.PMID 27525524Read ↗

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