Project Main Details
2013-05-28 13:05:56 GMT 2013-05-29 23:00:00 (GMT -05:00) Eastern Time (US & Canada) Yes (click here to learn more about ) Closed 0 0 0 direct invitation(s) have been sent by the voice seeker resulting in 0 audition(s) and/or proposal(s) so far. Voice123 SmartCast is seeking 50 auditions and/or proposals for this project (approx.) Invitations sent by SmartCast have resulted in 0 audition(s) and/or proposal(s) so far.
• Audio files must be delivered via FTP/Dropbox/Google Drive/cloud
The most severe form of the disease –
beta-thalassemia major – is fatal if not treated with frequent and lifelong blood transfusions.
The study is exploring a potential treatment option for those living with beta-thalassemia major.
The purpose of the study is to evaluate an investigational gene therapy approach - known as gene transfer - that may reduce or eliminate the need for blood transfusions and iron chelation therapy, as well as provide an alternative to allogeneic HSCT.
Beta-thalassemia is caused by a malfunction in the gene that makes beta-globin – a protein that is necessary for normal red blood cells.
In the study, patients’ blood stem cells will be collected to allow a functioning copy of the beta-globin gene to be transferred into them. The blood stem cells are collected in advance and the transfer of the functioning copy of the gene takes place outside the body, in a laboratory.
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