UMass Medical School announces ALS breakthrough

New England Council Member University of Massachusett’s Medical School and Massachusetts General Hospital recently announced a new breakthrough in amyotrophic lateral sclerosis (ALS) research. Researchers have developed a therapy that suppresses a harmful gene that causes that nervous system disease. The advancement is the latest made by UMass Medical School researchers, who have also identified genes critical to the development of ALS.   

UMass Medical School researchers have developed a method to reverse the damage caused by a gene called SOD1, which carries a genetic defect that causes ALS. In the lab, researchers have been able to attack the SOD1 gene with an altered virus, which has caused the proteins released by the gene to be far less harmful. According to UMass Medical School, the researchers are the first to safely treat two research participants with synthetic microRNA. The study was led by UMass researchers Dr. Robert Brown Jr. and Dr. Christian Mueller in collaboration with Dr. Merit Cudkowicz and Dr. James Berry at Mass General Hospital. Apic Bio, a company founded by Brown and Mueller, has licensed the discovery for clinical trials.   

“The biggest takeaway from this study is that we delivered a new class of silencing gene therapy to patients and suppressed levels of the ALS gene SOD1 quite effectively,” Dr. Mueller said in a statement.  

The New England Council commends UMass Medical School and Mass General for their discovery and progress towards a cure for ALS. Read more in the Worcester Business Journal

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