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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

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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 inserted. The...

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Gene therapy for sickle cell disease.

Abiola Olowoyeye¹, Charles I Okwundu

¹Lagos University TeachingHospital, Lagos, Nigeria. aolowoyeye@hotmail.com

The Cochrane Database of Systematic Reviews

|November 16, 2012

Summary

Gene therapy for sickle cell disease (SCD) shows promise, but no clinical trials have been reported. Further research is needed to evaluate its safety and effectiveness in SCD patients.

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 potential candidates for gene therapy due to the possibility of restoring normal function with a single gene copy.

Purpose of the Study:

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

Main Methods:

Searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register.
Included all randomized or quasi-randomized clinical trials (phases 1, 2, or 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 in sickle cell disease.