Reimaging beta-thalassemia and its treatment

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Beta-thalassemia is one of the world’s most common blood disorders. Scientists at St. Jude Children’s Research Hospital noticed it shares similarities with brain disorders like Alzheimer’s disease. Both involve abnormal proteins that build-up in cells.

The researchers turned that observation into a possible treatment approach. They found a drug called rapamycin that’s widely used to treat other diseases. This drug is also used with organ transplant patients. In the lab, researchers showed the drug eased symptoms. The drug also helped reduce the build-up of toxic proteins in developing blood cells from people with beta-thalassemia.

● Read the study press release:
● Read the Science Translational Medicine paper:
● Learn about the corresponding authors.
○ Mitchell Weiss, , , Department of Hematology chair:

○ Mondira Kundu, , , of the departments of Pathology and Cell & Molecular Biology:

● Follow @stjuderesearch across social media to learn about St. Jude science and medicine.
● Discover how you can join the St. Jude team:
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