Diseases, Conditions, Syndromes

New study shows promising evidence for sickle cell gene therapy

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).

Genetics

Researchers refine understanding of fetal hemoglobin regulation

Researchers at Children's Hospital of Philadelphia (CHOP) have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells. The findings, published today in Nature Genetics, refine ...

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, ...

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