Ribozymes To The Rescue: New UF Gene Therapy Shows Promise For Treatment Of Inherited Blindness

GAINESVILLE---University of Florida researchers have designed a new genetic weapon that can--in laboratory animals--significantly slow progression of retinitis pigmentosa, a leading cause of inherited human blindness.

The weapon, a ribozyme manufactured in the laboratory, works by chopping up genetic messengers before they deliver the instructions of a mutant gene.

"This is the first case we know of where ribozymes have been used to correct a disease in an animal in a way that realistically could be used in humans," said Alfred S. Lewin, a professor of molecular genetics and microbiology in UF's College of Medicine. "We also believe this technique might be useful for other inherited diseases."

Lewin and William Hauswirth, a professor with dual appointments in ophthalmology, as well as molecular genetics and microbiology, are leading a team conducting further animal studies in preparation for human clinical trials, an estimated five years away.

About 1 in 3,000 Americans has retinitis pigmentosa, a degenerative disease whose symptoms generally first appear during adolescence. As the disorder progresses, night vision, peripheral vision and ultimately all sight can be lost. Currently, there is no way to halt the deterioration.

Since the UF research was published in the August issue of Nature Medicine, Lewin has heard from many other scientists who are working to apply "ribozyme rescue" to heart disease, neurological disorders and viral diseases.

"UF's work is a great example of how fundamental research, whose goal is to understand basic cellular processes, can lead to important advances that have a direct impact on human health," said John Burke, a professor of microbiology and molecular genetics at the University of Vermont.

UF's research builds on the Nobel Prize-winning discovery in other laboratories that ribonucleic acid, or RNA, can act as an enzyme, a substance that causes chemical reactions within cells. It is then known as a ri

Contact: Victoria White
University of Florida

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