BOSTON, Dec. 9 (UPI) -- Children's Hospital Boston scientists say a new genetic approach to sickle-cell disease therapy is showing promise in animal studies.
The researchers said they have discovered silencing a specific gene boosts production of a fetal form of hemoglobin in mice, potentially compensating for the defective adult hemoglobin that causes red blood cells to "sickle" and obstruct blood flow.
Currently, there are only a limited number of therapies available for sickle-cell disease, the most common U.S. inherited blood disorder, said Dr. Stuart Orkin, the study's senior author.
Shortly after birth, babies switch from producing the fetal form of hemoglobin -- the protein inside red blood cells that carries oxygen -- to producing the adult form, But the scientists said it's long been known people who retain the ability to produce fetal hemoglobin have much milder disease.
Previous studies showed a gene called BCL11A is involved in switching off fetal hemoglobin production in adults. Working with genetically engineered mice, the researchers explored whether that switch could be used to alleviate the disease.
The study found that it could in mice.
If the preliminary results hold up in human studies, inactivating BCL11A might also help patients with thalassemia, another blood disorder involving abnormal hemoglobin, Orkin said.
The study was presented by first author Jian Xu this week in New Orleans during the annual meeting of the American Society for Hematology.
