Treatment (Gene Therapy)

by Farhana

Gene Therapy for Thalassemia Major
An Introduction to Gene Therapy for Thalassemia Major
Thalassemia research scientists are working to develop a gene therapy that may offer a cure for thalassemia. They are currently looking at two gene therapy treatments:

•Beta-globin gene and stem cells
•Medications and fetal hemoglobin.

Gene Therapy for Thalassemia Major: Beta-Globin and Stem Cells
This type of gene therapy for thalassemia major might involve inserting a normal beta-globin gene (the gene that is abnormal in this disease) into the patient's stem cells, which are the immature bone marrow cells that are the precursors of all other cells in the blood.

Gene Therapy for Thalassemia Major: Medications and Fetal Hemoglobin
Another form of gene therapy for thalassemia major could involve using drugs or other methods to reactivate the patient's genes that produce fetal hemoglobin, which is the form of hemoglobin found in fetuses and newborns. Scientists are seeking ways to activate these genetic switches so that they can make the blood cells of individuals with beta thalassemia produce more fetal hemoglobin to make up for their deficiency of adult hemoglobin.

Scientists hope that spurring production of fetal hemoglobin will compensate for the patient's deficiency of adult hemoglobin.


For beta thalassemia(but not available yet)
Thalagen™: Gene Therapy Treatment for Thalassemia
Thalagen™ is the brand name for EGT's gene therapy treatment for beta thalassemia, also referred to as Cooley's anemia. To date there is no curative therapy for thalassemia, a disease characterized by the cells of the bone marrow having an inability to produce normal hemoglobin. Currently, patients are treated with transfusion therapy, which aims to correct the anemia, suppress massive erythropoiesis and inhibit gastrointestinal absorption of iron. However, transfusion therapy worsens the iron overload, which over time is lethal if not treated.
Gene therapy is considered by most expert blood specialists as the most promising, long-term and cost-effective treatment of thalassemia. The objective of gene therapy is to insert the "normal" gene for hemoglobin into the DNA of the patient's bone marrow cells. The putative treatment procedure requires the collection of bone marrow hematopoietic stem cells from the thalassemic patient in the hospital followed by the treatment of these cells in the laboratory with the virus vector containing the gene for the production of normal hemoglobin. The treated bone marrow cells are then returned to the patient to begin the production of red blood cells with normal hemoglobin.
The proprietary EGT technology of Thalagen™ is anticipated to be:
•Erythroid-specific for elevated expression of the inserted human β-globin gene
•Long-term, producing sustained expression of the human β-globin gene
This method has been approved in April 2007.

Links:
1. http://blood.emedtv.com/thalassemia-major/gene-therapy-for-thalassemia-major.html
2. http://organizedwisdom.com/helpbar/index.html?return=http://organizedwisdom.com/Gene_Therapy_for_Thalassemia&url=www.errantgene.com/Product_Pipeline-Thalagen.shtml
3. http://organizedwisdom.com/helpbar/index.html?return=http://organizedwisdom.com/Gene_Therapy_for_Thalassemia&url=www.marchofdimes.com/pnhec/4439_1229.asp#head8