Hybridon Corrects Gene For Beta-Thalassemia

- Hybridon, in conjunction with researchers at the University of North Carolina, has corrected gene function inside a cell nucleus using Hybridon's antisense technology. The oligonucleotide compound restored an abnormal splice site activated by a genetic mutation for the blood disorder beta-thalassemia. The protein expression required for the disease process is thought to be halted by binding the antisense compound to the complementary nucleic acid sequence on the gene and changing the messenger RNA function. Full results of the preclinical animal study were published in the November 12 issue of the Proceedings of the National Academy of Sciences.