Cell stem cellSep 7, 2026

Using precise gene editing to treat inherited blood disorders affecting β-globin in different genetic backgrounds

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Sep 8, 2026

Abstract

All four patients, including one with sickle cell disease, achieved hematopoietic recovery and discontinued red blood cell transfusions.

All patients demonstrated durable editing and sustained high-level fetal hemoglobin expression after more than 12 months.
No vaso-occlusive episodes occurred in the sickle cell disease patient.
No off-target mutations, malignancies, or deaths were observed in any patient.
These findings suggest the potential applicability of a transformer base editor for treating sickle cell disease and specific types of beta-thalassemia.

Simplified

β-Hemoglobinopathies are monogenic disorders. We previously applied a transformer base editor (tBE) to reactivate fetal hemoglobin (HbF) expression, and five Chinese transfusion-dependent β-thalassemia (TDT) patients achieved transfusion independence. However, the applicability of tBE to sickle cell disease (SCD) and genetically different TDT populations remained unknown. Here, we show a four-patient descriptive report from three trials, including one African SCD patient and three TDT patients carrying mutations common in South and Southeast Asia. All patients achieve hematopoietic recovery, and red blood cell transfusions are discontinued in all cases. After more than 12 months of follow-up, all patients show durable editing, sustained high-level pan-cellular HbF expression, and transfusion independence. No vaso-occlusive episodes occur in the SCD patient. No off-target mutations, malignancies, or deaths are observed. These initial results support the feasibility of applying tBE to treat SCD and the reported TDT genotypes, warranting broader evaluation across diverse populations. ClinicalTrials.gov identifiers are as follows: NCT06328764, NCT06065189, and NCT06565026.

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CRISPR Gene EditingIssue #54

Four blood disorder patients across three continents stopped needing transfusions after base editing

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Using precise gene editing to treat inherited blood disorders affecting β-globin in different genetic backgrounds

Abstract

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