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September 18, 2025Stem Cells Translational MedicineOpen Access

Hematopoietic stem cell therapy with gene modification to treat sickle cell disease

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Authors

JBJennifer BallABAllan BradleyALAnh‐Tuan Le

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Overview

Review highlights advancements in hematopoietic stem cell therapy and gene editing in sickle cell disease, suggesting new treatment pathways.

Key Points

  • Gene therapy shows promise as a potential one-time cure for sickle cell disease, reducing long-term treatment requirements.
  • In vivo HSC gene therapy could simplify delivery methods, potentially improving accessibility and application for patients.
  • Autologous HSC gene therapy effectively utilizes patients’ own cells through novel techniques like lentiviral gene addition.
  • This review presents various therapeutic strategies and clinical trials, providing insights into the future of sickle cell disease treatment.

Cite This Study

Ball et al. (2025) studied this question.

synapsesocial.com/papers/68d435e4713b0b5dfea75a07https://doi.org/10.1093/stcltm/szaf042
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Also Consider

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

  1. 1Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches2025 · 14 citations
  2. 2Gene Therapy for Sickle Cell Disease: No Donor, No Problem2025
  3. 3Research on Treatment Strategies for the Treatment of Sicklemia Disease2025
  4. 4Sickle Cell Disease: From Ancient Origins to Modern Breakthroughs in Gene Therapy2026
  5. 5In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers2025 · 1 citations