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Updated: Nov 28, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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Gene therapy for sickle cell disease.

Abiola Olowoyeye1, Charles I Okwundu2

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Gene therapy shows promise for sickle cell disease (SCD), an inherited blood disorder. However, no clinical trials have been reported, highlighting the need for research to assess its safety and effectiveness.

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Area of Science:

  • Medical Genetics
  • Hematology
  • Gene Therapy

Background:

  • Sickle cell disease (SCD) is a group of genetic blood disorders caused by at least one hemoglobin S (Hb S) allele.
  • Autosomal recessive disorders like SCD are potential candidates for gene therapy due to the possibility of restoring a normal phenotype with a single normal gene copy.

Purpose of the Study:

  • To evaluate if gene therapy can enhance survival and prevent symptoms and complications in individuals with sickle cell disease.
  • To weigh the risks associated with gene therapy against its potential long-term benefits for SCD patients.

Main Methods:

  • Searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register and online registries.
  • Included all randomized or quasi-randomized clinical trials (Phase 1, 2, or 3) of gene therapy for sickle cell disease.
  • Most recent search conducted on September 21, 2020.

Main Results:

  • No trials of gene therapy for sickle cell disease were identified in the search.
  • No relevant randomized or quasi-randomized clinical trials were reported.

Conclusions:

  • Currently, no objective conclusions or practical recommendations can be made regarding gene therapy for sickle cell disease.
  • This review underscores the critical need for well-designed, randomized controlled trials to determine the benefits and risks of gene therapy for SCD.