CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes

Collectively, our preclinical findings suggest that the generation of the HPFH3 genotype has the potential to significantly enhance HbF levels, offering a promising universal therapeutic strategy for treating both SCD and β-thalassemia.

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Publication Details

Journal
Stem Cell Research & Therapy
Published
2025-12-21
DOI
https://doi.org/10.1186/s13287-025-04582-1
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
Field-Weighted Citation Impact
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article

CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes

Stem Cell Research & Therapy
Hemoglobinopathies and Related Disorders
article

CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes

article en

Abstract

Collectively, our preclinical findings suggest that the generation of the HPFH3 genotype has the potential to significantly enhance HbF levels, offering a promising universal therapeutic strategy for treating both SCD and β-thalassemia.

Stem Cell Research & TherapyVol. 17(1)
Institute of Genomics and Integrative Biology (IN), Rajiv Gandhi Cancer Institute and Research Centre (IN), Regional Centre for Biotechnology (IN), RIKEN BioResource Research Center (JP), Cell Medica (Switzerland) (CH), Indian Institute of Technology Kanpur (IN), Academy of Scientific and Innovative Research (IN)
Openalex Percentile: Top 38%
Hemoglobinopathies and Related Disorders
DOIOpen Access PDFOpenAlex
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CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes · Stem Cell Research & Therapy (2025) | TGRS Research Map | TGRS