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Revolutionary gene therapy gives hope to tens of thousands of people with sickle cell disease, after researchers at the Dana-Farber / Boston Children's Cancer Treatment and Blood Disease Center found a way to switch the switch "in the red blood cells.
"We are following very closely, but we are optimistic about its results so far," said Dr. Erica Esrick, co-principal investigator of the clinical trial. "It's our hope for this study – be it a healing approach."
Red blood cells with sickle cell disease are rigid and deformed, which gives them the shape of sickle identification instead of the smooth, round shape of healthy cells. Rigid sickle cells can block blood vessels, causing a series of acute or chronic symptoms of sickle cell disease.
The physicians and researchers involved in the trial are taking blood stem cells from a patient who undergoes genetic modification for months to promote healthy fetal hemoglobin production in the newborn, not the sickle. As a result, sickle cells "return" to healthy hemoglobin.
Cells modified by the gene are then returned to the patient through an infusion. To do this, patients like Emmanuel "Manny" Johnson from Brockton must undergo hospitalization and chemotherapy treatment for one or two months.
"When Manny received the infusion of his own stem cells in May, it was an emotional day for the entire team," Esrick said. "It's an exciting time for patients, doctors and researchers in the sickle cell community, with potentially healing approaches such as gene therapy and gene editing."
The trial is the result of a collaboration between the Dana-Farber / Boston Children's Blood Cancer and Blood Disorders Center and was led by Center Chair Dr. David A. Williams.
Once the test has been tested on a larger number of adults with sickle cell disease, it will also be extended to adolescents and children.
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