Doctors hopeful breakthrough gene therapy will lead to a cure for sickle cell disease

Researchers in Boston are hopeful that a new experimental gene therapy will help people with sickle cell disease.

Sickle cell disease, caused by a mutation in one gene, affects tens of thousands of people. Their red blood cells become rigid, and due to their irregular shape, the cells can get stuck and block blood flow, causing pain and serious conditions.

A team of doctors and researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center recently held a clinical trial that was able to "flip the switch" in red blood cells, The Boston Herald reports. They removed a patient's blood stem cells, which went through several months of gene modification so they would be able to to produce fetal hemoglobin; found in newborns, this does not sickle. Through an infusion, the gene-modified cells were given back to the patient.

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The patient, an adult man, was hospitalized for more than a month and received chemotherapy treatment. Dr. Erica Esrick, co-principal investigator of the clinical trial, told The Boston Herald the team feels "optimistic about his results so far. That is our hope for this study — that it is a curative approach."