Genetics

Decoding sickle cell disease

When Lamarcus Jean visits the Hematology Clinic at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, he makes himself right at home. The 6-year-old, whom his mom, Stephanie, describes as "wise beyond his years," ...

Medical research

Making blood on demand: How far have we come?

The reconstitution of the blood system in humans holds great therapeutic potential to treat many disorders, like blood cancers, sickle-cell anemia and others. Successful reconstitution requires the transplantation and engraftment ...

Diseases, Conditions, Syndromes

A potential Diamond-Blackfan anemia treatment swims into view

Zebrafish, besides being popular in aquariums, make good stand-ins for studying human diseases. They share about 70 percent of their genes with humans, and can be studied at a mass scale, enabling scientists to test hundreds, ...

Genetics

2019: the year gene therapy came of age

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering—by editing her genome.

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