Discussion Forums > General Chatter
IF I HAD ONLY HAD ONE WISH
Sharmin:
Tariq,
That would be soooo cool!
I think the gene therapy will happen one day, it sounds like they are close and they just have to perfect their technique ??? ??? All they need is more funding!!!!!! Hopefully the trials will show some success so that they can get the funds they require.
:pray :pray :pray :pray :pray :pray :pray :pray :pray :pray
Sharmin
Sharmin:
http://www.nyas.org/ebriefreps/main.asp?intSubsectionID=2707
--- Quote ---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.
--- End quote ---
maryo0m:
star light star bright i wish i may i wish i might be cured from thal this very night :-\
haha nice friend yaaap an affordable treatment dont we all want that? :biggrin
Dori:
Sharmin, a french men with thal. (major?) have had gene therapy around may. Did you hear about that?
One wish is hard :-\
I think the treatment issue of Maryoom is the best (at the moment).
Sharmin:
Yes Peartree girl,
We have actually posted regarding that patient on this site:
http://www.thalassemiapatientsandfriends.com/index.php?topic=1573.0
I believe there is new information regarding this trial and the patient on the September 2008/CAF newsletter - which I have not had a chance to read yet.
I hope this works!
Sharmin
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