Discussion Forums > Working Towards a Cure

CRISPR therapy for Thalassemia

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catchR:
This is indeed interesting development. Thanks for posting this!!

Hope Scientist continue to work on this approach and find success soon...

sofear:
(About the August article!)

Here is a topic on reddit with a few more useful comments: http://www.reddit.com/r/Futurology/comments/2cppiq/two_mutations_in_the_haemoglobin_gene_of_a/


--- Quote ---The effective gene therapies we are likely to see applied in the clinic in the next 10-15 years will nearly all be related to blood (including immune) disorders, because circulating cells are easy to isolate, manipulate, and reintroduce.
--- End quote ---

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--- Quote ---A blood transfusion would just be like giving him some healthy blood. To make the patient actually start producing healthy red blood cells on his own I'm thinking more along the lines of a bone marrow transplant.
--- End quote ---

I thought BMT has already been atleast an option to cure thalassemia for a long time. What's the difference with the new methods? That a patient does not need a donor anymore?

Andy Battaglia:
BMT has the risk of graft vs host disease, which does cause mortality in some patients. This risk is not present in gene therapy because you don't receive any donated genetic material.

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