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More Good News from Bluebird. FDA Grants Breakthrough Therapy Designation.

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Canadian_Family:
Thanks for sharing Himanshu.

"We are using our genome editing technology to target a key genetic switch in a patient's own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults.  We know that elevated production of fetal globin can ameliorate disease symptoms of hemoglobinopathies such as beta-thalassemia.  We are also developing this strategy for sickle cell disease."

I am always a big fan of this technique as these gamma chains capable to producing fetal hemoglobin goes silent after six month of birth. This techinique excludes the need of chemotherapy (if I am not mistaken) releiving the patient from side effects of Chemo. This is much natural cure than anything else in progress.

Parin:
This is amazing! Thank you both Andy and Himanshu for posting.

Regards,
Ashutosh T

Pratik:
Wow,

2015 is a holy grain for Thalpals!

Himanshu, thanks so much for posting that!

Canadian Family, yes, it would be much appreciated as a patient to have therapy without having to undergo Chemo.

Best,

-P

Sharmin:
Thank you for sharing Andy and Himanshu,

I agree Canadian Family.  I believe that over time the use of chemotherapy will be tapered and reduced.  As the process is fine tuned and refined.

My greatest concern with gene therapy has always been chemotherapy - if it can be avoided or reduced it would be best. 

Such great things happening for thalassemia and sickle cells patients.  Praying for a cure for a soon.

Sharmin :grouphug

catchR:
This is really amazing new for all the thals, thanks Andy and Himanshu for sharing!!

I truly hope these treatments soon become success and be available to larger community....

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