Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.

doi:10.1182/bloodadvances.2019000820

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Article

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.

Blood advances - 12 Nov 2019

Métais Jean-Yves, Doerfler Phillip A, Mayuranathan Thiyagaraj, Bauer Daniel E, Fowler Stephanie C, Hsieh Matthew M, Katta Varun, Keriwala Sagar, Lazzarotto Cicera R, Luk Kevin, Neel Michael D, Perry S Scott, Peters Samuel T, Porter Shaina N, Ryu Byoung Y, Sharma Akshay, Shea Devlin, Tisdale John F, Uchida Naoya, Wolfe Scot A, Woodard Kaitly J, Wu Yuxuan, Yao Yu, Zeng Jing, Pruett-Miller Shondra, Tsai Shengdar Q, Weiss Mitchell J

Abstract excerpt

Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9-mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strategy for sickle cell disease (SCD) and β-thalassemia, although the optimal technical approaches and limiting toxicities are not yet fully defined. We disrupted an HBG1/HBG2 gene...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 31698466
DOI: 10.1182/bloodadvances.2019000820
PMCID: PMC6855127

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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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