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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 inserted. The...

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 inserted. The...

Multiple Allele Traits

Multiple Allele Traits

The Concept of Multiple Allelism

iPS Cell Differentiation

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.

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.
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...

Bone Marrow Sampling and Transplants

Bone Marrow Sampling and Transplants

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.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...

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Related Experiment Video

Updated: Jun 10, 2026

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

Gene therapy for sickle cell disease.

Abiola Olowoyeye¹, Charles I Okwundu

¹Lagos University Teaching Hospital, P.O.Box 8893 Marina, Lagos, Nigeria.

The Cochrane Database of Systematic Reviews

|August 6, 2010

Summary

Gene therapy shows promise for sickle cell disease (SCD), an inherited blood disorder. However, current research is limited, with no clinical trials reported to date. Further studies are needed to assess its safety and effectiveness.

Area of Science:

Medical Genetics
Hematology
Gene Therapy

Background:

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

Purpose of the Study:

To evaluate if gene therapy can improve survival and prevent symptoms and complications in sickle cell disease patients.
To assess the risks versus potential long-term benefits of gene therapy for individuals with SCD.

Main Methods:

Searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register up to March 5, 2010.
Included all randomized or quasi-randomized clinical trials (Phase 1, 2, or 3) of gene therapy for sickle cell disease.

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Last Updated: Jun 10, 2026

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Main Results:

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

Conclusions:

No randomized or quasi-randomized clinical trials on gene therapy for sickle cell disease were found.
Objective conclusions or clinical recommendations cannot be made at this time.
There is a critical need for well-designed, randomized controlled trials to evaluate the benefits and risks of gene therapy for sickle cell disease.