UF reports possible breakthrough on treating macular degeneration


Published: Saturday, August 1, 2009 at 5:30 p.m.
Last Modified: Saturday, August 1, 2009 at 5:30 p.m.

A new approach to repairing damaged retinas in mice offers a ray of hope for some two million Americans with an age-related eye condition called macular degeneration.

University of Florida researchers report that they were able to program adult stem cells from mice to transform themselves into vision cells, suggesting a potential treatment for one of the most common causes of vision loss in older people.

In a paper to be published in September's Molecular Therapy, scientists describe how they used a virus carrying a gene that gently pushed cultured adult stem cells toward a fate as retinal cells. When the cultured cells were reintroduced into the mice, they were completely transformed into the desired type of vision cells.

"To our knowledge, this is the first reported use of targeted gene manipulation to specifically program an adult stem cell to become a new cell type," said Dr. Maria Grant, professor of pharmacology and therapeutics in the UF College of Medicine.

Ultimately, Grant said, the findings suggest that the same thing could be done with drugs.

"You would not give the drugs to the patient," she explained, "you would give the drugs to their cells. Take the cells out, activate certain chemical pathways, then put the cells back into the patient."

The researchers were able to use chemical compounds that mirrored environmental conditions in the body to point the stem cells toward their ultimate identities as vision cells.

In essence, they were successful in tricking the stem cell into thinking it is a retinal cell and behaving accordingly.

"This implies a whole new field of stem cell research that uses drug manipulation rather than genetic manipulation to send these immature cells along a new pathway," said Grant.

She collaborated in the work with Edward Scott, director of the program in stem cell biology and regenerative medicine at UF's McKnight Brain Institute.

"This work applies to 85 percent of patients who have age-related macular degeneration," Grant said. "There are no therapies for this devastating disease."

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