Gene Transfer
While stem cell transplantation has a high success rate among young, HLA-matched patients, researchers hope to find a more universally available cure. Gene transfer may be the solution. "The goal is to introduce a gene to restore normal function in red blood cells, removing the transfusion requirement," said Michel Sadelain, a researcher at Memorial Sloan-Kettering Cancer Center.
"The goal is to introduce a gene to restore normal function in red blood cells."
The process involves removing some of the patient's stem cells, inserting a gene that codes for the healthy globins, and then re-injecting the cells into the body. To introduce the new gene, Sadelain and his team have created a vector based on a lentivirus that is capable of introducing β-hemoglobin genes into the cells. They demonstrated the concept could work in thalassemic mice in 2003. "These mice remained alive only due to transplanted gene," said Sadelain. In the laboratory, they've shown that the gene vector works at least as well in human cells as it does in mouse cells, yielding a level of expression of about 55%.
A clinical trial of gene therapy to cure thalassemia is planned to begin in the next year in France, reported Arthur Bank, a physician and geneticist at Columbia University in New York and Philippe Leboulch at Harvard Medical School and Brigham & Women's Hospital in Boston. The first phase will enroll five patients with β thalassemia and look primarily at safety.
Gene therapy for a rare type of inherited blindness improved the vision of 4 adult patients!!
Success was reported by two separate teams of scientists treating Leber congenital amaurosis (LCA). As reported by the National Post in Canada.
A common cold virus (RPE65) was used to deliver the normal version of the gene into one eye of each patient (the other eye was used as a control). The patients went from almost complete blindness (only being able to detect hand movement) to actually reading lines on an eye chart with the treated eye.
These patients were not expected to improve much - because they already had significant damage and were given only low doses to test for safety of the procedure. Surprisingly, the gene therapy actually provided benefit for them.
This is great news for patients with LCA, gene therapy and anyone with a genetic disorder. I hope that this increases confidence in gene therapy!!
We received the CAF newsletter yesterday. There it talked about Gene therapy trials starting in US very soon. The newsletter also talked about the gene therapy trail going on in Paris. I forgot the name of the Doctor but the company associated with the trial is GENETIX Pharmaceuticals from cambridge, MA. The teenager who participated is doing well and continue to make some Hgb after the therapy. It did not mention if he is free from transfusion but it did mention that they already have another patient identified for theie gene therapy trial. So, may be we don't have to wait next 30 years for gene therapy to be available for our children