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Gene therapy cures boy of sickle cell disease

01-Mar-2017

Key points from article :

People with sickle cell disease make abnormal versions of haemoglobin – distorting red blood cells into a sickle-shape that can get stuck in blood vessels.

Bone marrow stem cells removed, treated, and replaced in boy’s body.

Medication and symptom free two years later.

Team has treated seven other patients, who are showing “promising” progress.

Treatment could work for everyone with the disorder.

The study was published in the New England Journal of Medicine.

Mentioned in this article:

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Deborah Gill

Professor of Gene Medicine at the University of Oxford.

Marina Cavazzana

Co-Founder and Acting CMO at Smart Immune, hematopoietic stem cell-based biotherapies

Stuart Orkin

Professor at Harvard Medical School.

The New England Journal of Medicine

Scientific Journal devoted to medical research.

Topics mentioned on this page:
Gene Therapy