News

Researchers at Dana-Farber/Boston Children's and UMass Medical School optimize gene editing for sickle cell disease and beta thalassemia

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases, sickle cell disease and beta thalassemia, applying CRISPR-Cas9 gene editing to patients' own blood stem cells.

Boston Children’s Hospital to receive $1.5M to initiate basic work to expand gene therapy treatment of sickle cell disease into developing countries

The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.

New gene therapy strategy for sickle cell disease shows early promise in humans

A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.

Zeroing in on the fetal-to-adult hemoglobin switch and a new way to combat sickle cell disease

In this Vector report, researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reveal how the protein, BCL11A, controls the switch in the body’s production of fetal hemoglobin to adult hemoglobin.

New gene therapy clinical trial offers hope for sickle cell disease

David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

Dana-Farber/Boston Children’s researchers, who are developing a gene therapy trial for sickle cell disease, commented in Stat and other outlets on a case report from a different trial.

Sickle cell disease: An unpredictable adversary

One teenage girl describes the intense pain of a sickle cell disease crisis as the feeling that heavy books are piled on her. Her sister likens it to being stabbed by needles. They don’t know when a debilitating crisis will strike, only that it will and that the sudden bouts of pain can last days, The Boston Globe reports.

Gene therapy offers hope for treatment of sickle cell anemia

Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in 2017.

We’ve known for 50 years what causes sickle cell disease. Where’s the cure?

Boston Globe Media’s Stat examines efforts to use gene therapy or gene editing – or develop a pill – to harness the latest research on sickle cell disease. “This is the right time for a sickle cell moonshot,” says Dana-Farber/Boston Children’s President David Williams, MD.