Sleeping Beauty 'Jumping Gene' Shows Promise For Sickle Cell Gene Therapy

In the study, scheduled for the June 12 issue of ACS' Biochemistry, a weekly journal, Clifford J. Steer and colleagues note that viruses have gotten most attention as possible vectors, or delivery vehicles, for replacing defective genes with normal copies.

In SCD, a mutation in the gene that encodes for beta globin results in abnormal hemoglobin that gives red blood cells a sickle shape. Concerns about potential risks and other problems with viral vectors, however, have become barriers to use of gene therapy.

Using laboratory cell cultures, the researchers showed that SB-Tn system could transfer normal beta globin genes into cells. The system, named for a fish gene reawakened by other researchers in 1997 after 15 million years of dormancy, fulfills essential requirements for gene therapy, the report states. Cells take up genes transferred with SB-Tn technology, the genes produce beta globin in stable fashion for long periods, and the genes are inherited and passed along as cells reproduce.

Scientists term Sleeping Beauty as a transposon, or a "jumping gene" because it can jump from one location on a piece of DNA to another.