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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Related Experiment Video

Updated: Mar 6, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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Rounding up sickle cells with gene therapy.

Leah Byrne1

  • 1Department of Ophthalmology, University of Pittsburgh, Pittsburgh, PA 15213, USA.

Science Translational Medicine
|March 17, 2017
PubMed
Summary

Gene therapy using ex vivo lentiviral gene transfer to hematopoietic stem cells offers promising treatment for sickle cell anemia patients. This innovative approach shows potential for managing this complex genetic blood disorder.

Area of Science:

  • Hematology
  • Gene Therapy
  • Molecular Biology

Background:

  • Sickle cell anemia is a debilitating inherited blood disorder.
  • Current treatments for sickle cell anemia have limitations.

Purpose of the Study:

  • To report on a patient treated with ex vivo lentiviral gene transfer.
  • To demonstrate the potential of gene therapy for sickle cell anemia.

Main Methods:

  • Ex vivo lentiviral gene transfer was performed on hematopoietic stem cells.
  • The modified cells were administered to a patient with sickle cell anemia.

Main Results:

  • The treatment showed promise in managing sickle cell anemia.
  • Successful gene transfer to hematopoietic stem cells was achieved.

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Conclusions:

  • Ex vivo lentiviral gene transfer is a viable therapeutic strategy for sickle cell anemia.
  • Gene therapy holds significant promise for the future treatment of sickle cell anemia.