New Agent To Fight Genetic Disorders Found

May 1, 2007

A new agent, called " Zorro-LNA, " appears to have the potential to

stop genetic disorders in their tracks. In a study to appear in the

June 2007 issue of The Federation of American Societies for

Experimental Biology Journal, 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 Edvard 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 significantly raise the

possibility that new therapies could arise where defective DNA is

deactivated more completely and more thoroughly than ever before.

For instance, Zorro-LNA could be used in combination with " RNA

interference " (RNAi). Like Zorro-LNA, RNAi has the ability to

deactivate genes, but does so by degrading the gene's RNA. In

addition, Zorro-LNA could be used to deactivate certain genes in

stem cells, which could eventually lead to the development of new

cells, tissues, or organs. (The discovery of RNAi was recognized by

a Nobel Prize award in 2006 to two American scientists.)

" This is a major development in the treatment not only of genetic

diseases, but also of acquired diseases when microbes or toxins

cause genes to go awry " said Gerald Weissmann, M.D., Editor-in-Chief

of The FASEB Journal. " One might say these researchers have found a

gene-hunter's Holy Grail for which scientists have been hunting for

many years. Zorro-LNA should give us a new, safe way of blocking the

effects of errors in our genetic repertoire. "

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