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August 22, 2025Frontiers in PharmacologyOpen Access

Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches

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Authors

MTMuhammad TaherSASofea ‘Aisyah AminondinNNNur Asyilah Nasir

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Overview

This review highlights gene therapy's role and hematopoietic stem cell transplantation in sickle cell disease management, suggesting new options.

Key Points

  • Gene therapy shows promise in treating sickle cell disease, preventing hemoglobin aggregation and improving symptoms.
  • Clinical trials indicate that lentiviral vector-mediated gene insertion offers significant therapeutic benefits for patients.
  • Allogeneic hematopoietic stem cell transplantation can alleviate symptoms but faces donor shortages and immunological challenges.
  • Emerging CRISPR/Cas9 technology represents a shift towards practical gene editing solutions for sickle cell disease.

Cite This Study

Taher et al. (2025) studied this question.

synapsesocial.com/papers/68af73637567bf4f94fed2f5https://doi.org/10.3389/fphar.2025.1630994
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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: From Ancient Origins to Modern Breakthroughs in Gene Therapy2026
  2. 2Hematopoietic stem cell therapy with gene modification to treat sickle cell disease2025
  3. 3Research on Treatment Strategies for the Treatment of Sicklemia Disease2025
  4. 4Gene Therapy for Sickle Cell Disease: No Donor, No Problem2025
  5. 5Antiretroviral Therapy and Sickle Cell Disease: Balancing Efficacy and Toxicity2025