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ALS Worldwide
April 17, 2017

Brilliant Find by Mayo Clinic Should Help Develop New Therapies For ALS

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ALS doesn't have a cure, but a Mayo Clinic team found a protein that could lead to development of new therapies.

ALS affects motor neurons, which transmit messages from the brain or spinal cord to muscles or glands. As the disease progresses, patients lose the ability to move their muscles. The Mayo Clinic team looked at a mutation of the gene C9ORF72, the most common genetic cause of ALS. They found that the mutation was responsible for the buildup of a protein called polyGP in the cerebrospinal fluid and blood cells.

They located polyGP in the cerebrospinal fluid of 134 people carrying the C9ORF72 mutation, including 83 people with ALS. The protein was absent in 120 people who did not have the mutation, including those with a different type of ALS. The team then injected a drug targeting the mutation into the brains of mouse models of ALS. After eight weeks, they saw a “marked decrease” in polyGP levels and a reduction in mRNA carrying the mutation. Testing polyGP levels in cerebrospinal fluid could influence the development of ALS drugs."

To read the full article, please visit http://www.fiercebiotech.com/…/new-marker-could-break-als-d….