A study to appear in the June 2007 issue of The FASEB Journal describes a new agent, called Zorro-LNA, which has the potential to stop genetic disorders in their tracks.
In the study, researchers from the Karolinska Institute in Stockholm, Sweden, describe how they developed Zorro-LNA to bind with both strands of a gene’s DNA simultaneously, effectively disabling that gene.
This development has clinical implications for virtually every human condition caused by or worsened by dominant defective genes. Examples include: Huntington’s disease, familial high cholesterol, polycystic kidney disease, some instances of glaucoma and colorectal cancer, and neurofibromatosis, among others.
"Zorro-LNA is a new substance that targets DNA and turns off genes," said co-author Edva Smith of the Karolinska Institute in Sweden. "It has the potential of becoming a new drug for the treatment of human genetic disease."
The findings described in this article raise the possibility that new therapies could arise where defective DNA is deactivated more completely and more thoroughly than ever before.