UF researchers reverse disease in mice


University of Florida researchers found a gene therapy that reversed and prevented multiple sclerosis in mice, according to a study published Thursday in the journal Molecular Therapy.

University of Florida researchers have found a way to reverse and prevent multiple sclerosis in mice, according to an article published Thursday in the journal Molecular Therapy.

It might be three to five years before the research makes its way into clinical labs for humans, said Brad Hoffman, an associate professor in the departments of pediatrics and neuroscience at the UF College of Medicine. But Hoffman said he hopes this will further development.

“This is a new avenue of gene therapy,” Hoffman said. “It’s setting a foundation of exploring for further research.”

The therapy combines the gene of a brain protein, myelin oligodendrocyte glycoprotein, and the drug Rapamycin, which coats heart stents and prevents organ transplant rejection.

Multiple sclerosis occurs when the immune system attacks the myelin sheath that surrounds and protects nerve fibers, which then leads to muscle weakness and problems in vision, speech and muscle coordination.

In the study, myelin oligodendrocyte glycoprotein was delivered to mice’s livers. The protein increased production of regulatory T cells, which suppress those immune system cells that attack the sheath.

Hoffman said the study showed that when the gene therapy was an early intervention, many mice recovered from the multiple sclerosis. Of mice with partial paralysis, 80 percent showed stronger mobility days after receiving the gene therapy. Mice who received the therapy had no signs of the disease after seven months.

Mice in a control group showed neurological problems 14 days later.

“It’s a very strong and potent result that we get from that treatment,” Hoffman said.

The treatment was a decade in the making, and similar therapies have been developed for other diseases, Hoffman said.

Hoffman said he’s cautiously optimistic about eventually using the treatment in humans.

“This is done in a mouse model, and we always have to take that with a grain of salt,” he said.



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