Discussion Forums > Working Towards a Cure
Bluebird Bio Releases Data on Current Gene Therapy Trials
Andy Battaglia:
No problem. Just realize that I am an incredibly busy person even before I get to thal work, so I do have to often put things off until I have more time. My activity on thalpal does normally increase on weekends when I am not at work. Lately, my efforts have been reduced by illness. Hopefully, this lingering flu will soon clear up. I hate to respond to many posts when my mind is not as clear as I wish. If I feel I cannot adequately process thought, I will wait until my head clears up.
jay:
How gene therapy works? TEDx by a Bluebird guy
https://www.youtube.com/watch?v=Ez560GnkSrE
jay:
I have been studied biology till age of 19 before moving on to technology, I can do some basic DNA tests, I can do electrophoresis, and I think I understood the explanation in previous post. (These all 'I can's where unnecessary). But again I stuck, pity on me, do this process cause additional mutation in the particular cells? Do this process cause extra addition in genome other that the mutated part? Are there other parts of vector grow along with the cells?
Andy Battaglia:
No, no mutation is caused. Working beta globin genes are inserted into the patient where they can multiply. The gene vectors are produced in a lab and are not from a donor, so no reactions. To put it simply, something is added that is not already there. It's a normally working gene. The method varies slightly between companies, as only the trial at Sloan Kettering uses the beta globin gene exactly as is found in the human body. The Northstar trial uses a mutated gene, but this causes no mutation in the body, anymore than the mutated gene already present in thals does not cause any mutation of anything else in the body. This is all confined to the beta globin gene. So, instead of relying on a mutation that does not work, they use a mutation that does produce beta globin. This new gene's output can thus be easily monitored and we see the results in the trial reports, as the new hemoglobin being produced is listed as part of the whole in the charts that have been posted. The new hemoglobin produced by the new gene is labeled as HbA t87Q in the charts.
Sharmin:
Andy,
Thank you kindly for synthesizing this information for us. I hope that you are starting to feel better! I was wondering if you had heard anything from Sloan Kettering.
Sharmin
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