Diseases, Conditions, Syndromes

Toward a reliable oral treatment for sickle cell disease

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...

Genetics

Addressing a complex world of pain in a single gene difference

A single letter difference in a single gene, inherited from both parents, spells a lifetime of anemia and pain for 20 million people, mostly of African ancestry, worldwide. Sickle cell disease (SCD) causes red blood cells ...

Genetics

Still too soon to try altering human embryo DNA, panel says

It's still too soon to try to make genetically edited babies because the science isn't advanced enough to ensure safety, says an international panel of experts who also mapped a pathway for any countries that want to consider ...

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