Unique Gene Therapy Prevents, Reverses Multiple Sclerosis in Animal Model
Multiple sclerosis can be inhibited or reversed using a novel gene therapy technique that stops the disease’s immune response in mouse models, University of Florida Health researchers have found.
This news is truly comforting!
By combining a brain-protein gene and an existing medication, the researchers were able to prevent the mouse version of multiple sclerosis. Likewise, the treatments produced near-complete remission in the animal models. The findings, which researchers said have significant potential for treating multiple sclerosis and other autoimmune disorders, were published Sept. 21 in the journal Molecular Therapy.
“Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,” said Brad E. Hoffman, Ph.D., an assistant professor in the departments of pediatrics and neuroscience at the University of Florida College of Medicine.
Before the therapy can be tested in humans during a clinical trial, further research involving other preclinical models will be needed, Hoffman said. Researchers also need to target the full suite of proteins that are implicated in multiple sclerosis, he added.
Still, Hoffman said he is extremely optimistic that the gene therapy can be effective in humans.
“If we can provide long-term remission for people and a long-term quality of life, that is a very promising outcome,” he said.
► Read the science paper “Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis”, published in Molecular Therapy >>