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posted by Fnord666 on Thursday April 19 2018, @03:04PM   Printer-friendly
from the positive-results dept.

Gene Therapy For Inherited Blood Disorder Reduced Transfusions

Gene therapy is showing promise for treating one of the most common genetic disorders. Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.

[...] The researchers stress, however, that more research is needed to fully evaluate how well the treatment works and how safe it is. Still, the company that's developing the treatment, Bluebird Bio of Cambridge, Mass., plans to seek approval of the treatment in Europe by the end of the year, a spokeswoman said in an email.

An estimated 288,000 people have beta-thalassemia worldwide, which makes the disease one of the most common genetic disorders. It's found most often in Mediterranean countries, the Middle East, Asia, India, and parts of Africa and South America. In the United States, about 10,000 to 15,000 patients have beta-thalassemia.

Also at BBC.

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia (DOI: 10.1056/NEJMoa1705342) (DX)


Original Submission

 
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  • (Score: 0) by Anonymous Coward on Thursday April 19 2018, @07:48PM (1 child)

    by Anonymous Coward on Thursday April 19 2018, @07:48PM (#669252)

    Looks like it's safe to say that no one on SN today has Beta-Thalassemia Blood Disorder. It might even be safe to say that no one has any relatives with it either...?

  • (Score: 2) by opinionated_science on Friday April 20 2018, @03:19PM

    by opinionated_science (4031) on Friday April 20 2018, @03:19PM (#669668)

    The hetero disorder (one bad copy of beta-globin, tagged Beta-thalassemia-minor) is not fatal, and has been shown to be slightly protective of malaria (due to reduced RBC size).

    Hence, many members of populations where malaria is commonplace, will have a chance of offspring carrying the double mutation, as the hetero-disorder may prove a slight advantage.

    Without modern medical help (transfusions/ chelyation therapy and the associate diagnostics), it is often fatal before adulthood.

    There are many disorders in this category, expect to see many new releases if this method is successful (e.g. Sickle cell etc..)