Helping Red Blood Cells Breathe Easier

New gene therapy technique which has the possibility of treating two common red blood cell disorders that affect millions of people world wide

A team of researchers from the Weill Cornell Medical College has designed a gene therapy that has the ability to treat both ß-thalassemia disease and sickle cell anemia, two of the most common genetic red blood cell disorders.  The technology available to them has also given them the ability to predict how the treatment will affect these diseased individuals.  This new approach to treating these red blood cells disorders has the potential to cure many patients even before they are affected with the disease.

ß-thalassemia is an inherited disease that neglects the production of the hemoglobin protein that carries oxygen throughout the body.  The new gene therapy technique developed ensures that the gene that is delivered is an active and working protein.  In order to achieve the transfer, the researchers generated lentiviral-mediated transfers of the human ß-globin gene to the bone marrow stem cells of the infected patients and then delivered back via a bone marrow transplant.  The stem cells would then produce healthy ß-globin protein and hemoglobin.

The insulator that they simulated produced two goals including the protection of the delivery of a normal ß-globin as well as increasing the efficiency at which the ß-globin gene is transcribed during the process of making red blood cells.  The vector that the research team created had different rates of efficiency based on the mutations in each patient.  Therefore, this provided the information for the team to predict which individuals would be the best candidates for these trails.

Among the 19 different samples they tested they found that on average the new cells produced 55% of the adult hemoglobin seen in unaffected individuals.

“This study represents a fresh departure from previously published work in the field of gene therapy,” Dr. Rivella says.  These results that the research team found will make an impact on the furthering of gene regulation and the transfer of genes for other gene therapy experiments.

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