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Related Concept Videos

Gene Therapy00:59

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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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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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Related Experiment Video

Updated: Mar 12, 2026

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 Okwundu

  • 1Children's Hospital Los Angeles, 1520 Rodney Drive apt 216, Los Angeles, California, USA.

The Cochrane Database of Systematic Reviews
|November 15, 2016
PubMed
Summary

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

Area of Science:

  • Hematology
  • Genetic Medicine
  • Gene Therapy

Background:

  • Sickle cell disease (SCD) is a group of genetic disorders involving the hemoglobin S (Hb S) allele.
  • Autosomal recessive disorders like SCD are candidates for gene therapy due to the potential to restore normal function with a single gene copy.

Purpose of the Study:

  • To determine if gene therapy improves survival and prevents complications in sickle cell disease patients.
  • To evaluate the risks versus potential long-term benefits of gene therapy for sickle cell disease.

Main Methods:

  • Searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register.
  • Included randomized or quasi-randomized clinical trials (Phase 1-3) of gene therapy for sickle cell disease.

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Main Results:

  • No trials of gene therapy for sickle cell disease were found.
  • No randomized or quasi-randomized clinical trials were reported.

Conclusions:

  • No objective conclusions or recommendations can be made regarding gene therapy for sickle cell disease.
  • There is a need for well-designed, randomized controlled trials to assess the benefits and risks of gene therapy for sickle cell disease.