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

Muhammad Taher1, Sofea 'Aisyah Aminondin1, Nur Asyilah Nasir1

  • 1Faculty of Pharmacy, International Islamic University Malaysia, Kuantan, Malaysia.

Frontiers in Pharmacology
|September 8, 2025
PubMed

Insights

Gene therapy offers a promising alternative for sickle cell disease (SCD) treatment. Autologous gene-modified stem cell transplantation effectively alleviates SCD symptoms without immune complications, advancing therapeutic options.

Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Sickle cell disease (SCD) is an inherited blood disorder caused by abnormal hemoglobin production, leading to red blood cell sickling.
  • A single point mutation in the β-globin chain causes deoxyhemoglobin aggregation, resulting in vaso-occlusive and hemolytic crises.

Purpose of the Study:

  • To review therapeutic advances for sickle cell disease (SCD).
  • To analyze recent developments in gene therapy for SCD treatment.

Main Methods:

  • Systematic review of articles published between January 2015 and January 2025.
  • Keyword-based search focusing on SCD and therapeutic advancements across three databases.

Main Results:

  • Allogeneic hematopoietic stem cell (HSC) transplantation shows efficacy but faces donor limitations and immunological issues.
  • Autologous gene-modified HSC transplantation via gene therapy provides similar benefits without immunological complications.
  • Lentiviral vector gene therapy and CRISPR/Cas9 gene editing demonstrate significant therapeutic potential in clinical trials.

Conclusions:

  • Gene therapy, particularly autologous HSC transplantation, represents a significant advancement in SCD treatment.
  • Gene editing technologies like CRISPR/Cas9 are transitioning SCD gene therapy from concept to clinical reality.
  • These advanced therapies offer improved outcomes and fewer complications for sickle cell disease patients.

Keywords:
CRISPRanemiagene editinggene therapyhematopoietic stem cell transplantationhemoglobinopathies

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