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Turning Blood Red: The Fight for Life in Cooley's Anemia

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Andy Battaglia:
Dr Bank,

I do have one question that I imagine is on the minds of most. From what has been seen with this patient, is there a belief that this process can eventually produce a higher output of beta globin? What has been accomplished here is a good first step, but for a beta zero patient, a bigger boost in hemoglobin would be necessary to free the patient from transfusions. Are there things, such as increasing the amount of mobilized stem cells that can be changed that would hopefully bring a significantly higher beta globin output?

arthurbank:
I believe that the globin gene therapy trial underway in Paris and those planned by others can produce the amount of hemoglobin necessary to allow patients with beta zero-beta zero thalassemia to become transfusion independent. This goal can be accomplished by increasing the number of gene-expressing stem cells and by increasing the amount of new normal hemoglobin produced in gene corrected red blood cells. 

arthurbank:
Hi everyone. I am attaching a copy of a recent review article I wrote on gene therapy for thalassemia in the Hematology/Oncology Clinics of North America.

I also wanted you all to know that my book, "Turning Blood Red: The Fight for Life in Cooley's Anemia," is now available on Amazon Kindle and on your iPad or iPhone through Apple's iMacBook store.

Arthur

Andy Battaglia:
Thank you Dr Bank. I have passed on the note about how to buy an E-copy of your book to our group on Facebook.

The summary you have provided is quite interesting and helps to demonstrate the progress being made with each new trial. The key does seem to be introducing enough of the beta globin gene into the patients to cause a large enough increase in beta globin production to free patients from transfusions. Recent progress in stem cell mobilization will prove to be quite significant for this process.

Dr Bank, do you feel that this same process can eventually be used to treat other genetic disorders? And if so, do you see this as one of the biggest developments in medicine ever?

arthurbank:
I'd like to join your group on Facebook, but couldn't find thalplal or you. Help!

Gene therapy is being used in several other diseases. Patients with subacute combined immunodeficiency (X-SCID) and adenosine deaminase deficiency (ADA deficiency) have been cured by gene therapy. In these diseases, relatively low levels of corrective gene expression lead to preferential survival of of gene-corrected cells. Patients with adrenoleukodystrophy (a neurologic condition), chronic granulomatous disease (a white blood cell disease). hemophilia, and Wiskott-Aldrich syndrome (a platelet disorder) have all been treated with gene therapy as well.

As far as your question about the role of gene therapy in medicine long-term is concerned, I would say that remains to be determined in the future.

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