Discussion Forums > Working Towards a Cure
Bluebird Bio Releases Data on Current Gene Therapy Trials
jay:
Hi Andy,
A question for Bluebird.
Will they release subject's full genome sequence public before and after gene therapy?
Is it the part of their terms and conditions with subjects?
I tried to search if putting such condition in trial agreement is allowed by US law but not successful.
jay:
So many questions to Andy, please answer anyone,
In addition to my previous post, after seeing David Levy's interview by Josephine, another question to Bluebird.
What is the exact mutation they cure? or do they replace entire HBB?
There are multiple Thalassemia major mutations on HBB.
Andy Battaglia:
Jay,
I have many urgent matters to deal with on a daily basis, so I must prioritize. Addressing situations that require immediate attention will always take precedence over questions that have no urgency. I hope you do realize that my efforts are volunteer and that I give an inordinate amount of time to thals. I have no appreciation for impatience. Excuse me if my time has been spent on helping people with urgent issues that should have been addressed by their own doctors long ago. I really will put the mother of a 36 year old woman whose life is not worth living above random questions that have absolutely no urgency. In addition to my public work here, I run three Facebook thal groups and am constantly inundated with emails and personal messages. It becomes quite frustrating when adults show a lack of patience. And I say all this while suffering for over two weeks from the flu, yet I have missed no work and have attended to some very urgent cases, in spite of feeling terrible.
To answer the questions, they are doing DNA testing of patients. This is seen in each patient being labeled HbE beta thal +, beta zero zero, beta + zero, etc. They can only know this through testing. The reason for doing this is to show that it works on all patients, regardless of the severity of their thalassemia. The original condition has no relevance in terms of the cure, though. Good beta globin genes are inserted into the body and what is already there has no relevance beyond its initial ability to produce hemoglobin, which in the end will be part of the whole. What trials will show is what has to be done in terms of preparation in terms of chemotherapy, and how dosing has to be adjusted for individual cases. The original genotype has no effect on this process. Unlike with BMT, you are not changing the patients genetically. It is simply insertion of working genes. Their function substitutes for what is already in the body and does not work properly. The old genes are still there and 'working" but a newly introduced beta globin gene will do the work. You can almost look at it as a recipe that needs something. Ah, salt! It's incomplete until salt is added and the blood making ability of thals is incomplete until you add the salt of a working gene and now the recipe works.
Andy Battaglia:
And Jay, if I have missed any questions with my responses, please point to the posts and I will address therm. I try to catch up on weekends when time permits, and try to find everything I have missed. but sometimes overlook posts.
jay:
Hi Andy,
I must apologize for sounding urgent, I am not native English, and actually I wanted someone else to answer it because I do understand the extra efforts you put and I am aware of it from long time. This forum has given immense knowledge to me and I couldn't have done better it without this forum. I feel so many questions are directed to you because everybody believes in you. :imsorry
Navigation
[0] Message Index
[#] Next page
[*] Previous page
Go to full version