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Gene Therapy

Gene Therapy

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...

Gene Therapy

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Stem Cell Therapy for Tissue Regeneration

Stem Cell Therapy for Tissue Regeneration

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: Apr 22, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy

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

|October 11, 2014

Summary

Gene therapy for sickle cell disease (SCD) is promising but lacks clinical trials. Further research is needed to assess its safety and effectiveness in managing SCD complications.

Area of Science:

Medical Genetics
Hematology
Gene Therapy

Background:

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

Purpose of the Study:

To evaluate if gene therapy improves survival and prevents symptoms/complications in sickle cell disease patients.
To assess the risks versus 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 all randomized or quasi-randomized clinical trials of gene therapy for sickle cell disease.

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Main Results:

No trials of gene therapy for sickle cell disease were identified in the search.
No reported trials mean no data on the efficacy or safety of gene therapy for SCD.

Conclusions:

No randomized or quasi-randomized clinical trials for gene therapy in sickle cell disease were found.
Objective conclusions or recommendations for gene therapy in SCD cannot be made.
There is a need for well-designed, randomized controlled trials to evaluate gene therapy for sickle cell disease.