Unique Gene Therapy Prevents, Reverses Multiple Sclerosis in Animal Model

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.

Learn more at UF NEWS>> http://news.ufl.edu/articles/2017/09/unique-gene-therapy-prevents-reverses-multiple-sclerosis-in-animal-model.php

► 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 >>
http://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(17)30413-6

#Neuroscience, #Research, #MultipleSclerosis, #GeneTherapy, #Brain, #MouseModels

Researchers make major brain repair discovery in fight against Multiple Sclerosis

Researchers make major brain repair discovery in fight against Multiple Sclerosis
Queen’s University Belfast scientists have discovered that specific cells from the immune system are key players in brain repair.

The research study, led by Dr Yvonne Dombrowski and Dr Denise Fitzgerald at the Wellcome-Wolfson Institute for Experimental Medicine at Queen’s University Belfast, is being hailed as a landmark study in unravelling the mysteries of how the brain repairs damage. This is crucial in the fight against MS, which affects 2.3 million people world-wide and over 4,500 people in Northern Ireland.

MS is the most common neurological disease affecting young adults and is the result of damage to myelin, the protective sheath surrounding nerve fibres of the central nervous system – the brain, spinal cord and optic nerve. In MS, the immune system wrongly attacks the myelin sheath covering nerve fibres in the brain and spinal cord, which can lead to symptoms such as vision loss, pain, fatigue and paralysis.

Until now, medical treatment could limit relapses but could not reverse the damage already done by the condition. The exciting aspect of this new research is that the team have uncovered beneficial effects of immune cells in myelin repair that have potential to reverse myelin damage. The study was an international collaboration including experts in Cambridge, San Francisco, Edinburgh, Maynooth and Nice.

This is a fundamental breakthrough that could revolutionise the treatment of debilitating neurological disorders such as Multiple Sclerosis (MS).

The research breakthrough, which has been published in Nature Neuroscience, shows that a protein made by certain cells within the immune system triggers the brain’s stem cells to mature into oligodendrocytes that repair myelin.

The discovery means that researchers can now use this new knowledge to develop medicines which will boost these particular cells and develop an entirely new class of treatments for the future.

Source & further reading:
http://www.qub.ac.uk/Connect/News/Allnews/QueensUniversityresearchersmakemajorbrainrepairdiscoveryinfightagainstMultipleSclerosis.html

Journal article:
http://www.nature.com/neuro/journal/v20/n5/full/nn.4528.html?foxtrotcallback=true

#neuroscience #MS #immunesystem #myelin #oligodendrocytes #immunecells #research