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December 8, 2025BloodOpen Access

In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers

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Authors

GSGiulia SchiroliMMMichael T. MonteNBNouhaila Beytour

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Overview

RNA gene writers effectively edit the HBB gene in hematopoietic stem cells, suggesting a safer treatment for sickle cell disease.

Key Points

  • This study aims to correct the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers.
  • Applied RNA gene writers to correct the HBB E6V mutation in hematopoietic stem cells.
  • Utilized intravenous lipid nanoparticles for delivery of RNA-based gene editors.
  • Conducted in vivo editing in both humanized mice and non-human primate models.
  • Achieved 70% correction of HBB mutation in SCD patient-derived mouse models.
  • Demonstrated durable editing levels confirmed by stable multi-lineage engraftment post-transplantation.
  • Restored adult hemoglobin levels by 98% in corrected cells undergoing erythroid differentiation.

Cite This Study

Schiroli et al. (2025) studied this question.

synapsesocial.com/papers/69362f3d4fa91c937236d490https://doi.org/10.1182/blood-2025-2536
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1In Vivo RNA delivery by targeted lipid nanoparticles enable gene editing in hematopoietic stem cells and T cells2025 · 5 citations
  2. 2Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models2021 · 27 citations
  3. 3Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model2025 · 1 citations
  4. 4Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells2024 · 20 citations
  5. 5New approaches in gene therapy for sickle cell disease, moving in vivo2024